부산대학교 도서관

Innes, Emily A ; Marne, Fleur Annette Le ; Macintosh, Rebecca ; Nevin, Suzanne M. ; Briggs, Nancy E ; Vivekanandarajah, Sinthu ; Webster, Richard I ; Sachdev, Rani K ; Bye, Ann M E

In Seizure: European Journal of Epilepsy February 2024 115:1-13

Ng, Bobby G; Eklund, Erik A; Shiryaev, Sergey A; Dong, Yin Y; Abbott, Mary‐Alice; Asteggiano, Carla; Bamshad, Michael J; Barr, Eileen; Bernstein, Jonathan A; Chelakkadan, Shabeed; Christodoulou, John; Chung, Wendy K; Ciliberto, Michael A; Cousin, Janice; Gardiner, Fiona; Ghosh, Suman; Graf, William D; Grunewald, Stephanie; Hammond, Katherine; Hauser, Natalie S; Hoganson, George E; Houck, Kimberly M; Kohler, Jennefer N; Morava, Eva; Larson, Austin A; Liu, Pengfei; Madathil, Sujana; McCormack, Colleen; Meeks, Naomi JL; Miller, Rebecca; Monaghan, Kristin G; Nickerson, Deborah A; Palculict, Timothy Blake; Papazoglu, Gabriela Magali; Pletcher, Beth A; Scheffer, Ingrid E; Schenone, Andrea Beatriz; Schnur, Rhonda E; Si, Yue; Rowe, Leah J; Russi, Alvaro H Serrano; Russo, Rossana Sanchez; Thabet, Farouq; Tuite, Allysa; Villanueva, María Mercedes; Wang, Raymond Y; Webster, Richard I; Wilson, Dorcas; Zalan, Alice; Network, University of Washington Center for Mendelian Genomics Undiagnosed Diseases; Wolfe, Lynne A; Rosenfeld, Jill A; Rhodes, Lindsay; Freeze, Hudson H

Journal of Inherited Metabolic Disease, vol 43, iss 6

Briest RC; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; Cheung AK; Department of Neurointervention, Prince of Wales Hospital, Randwick, NSW, Australia.; Kandula T; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; School of Women's and Children's Health, University of New South Wales, Sydney, NSW, Australia.; Cardamone M; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; School of Women's and Children's Health, University of New South Wales, Sydney, NSW, Australia.; Pillai SC; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; School of Women's and Children's Health, University of New South Wales, Sydney, NSW, Australia.; Sampaio H; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; School of Women's and Children's Health, University of New South Wales, Sydney, NSW, Australia.; Teoh HL; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.; School of Women's and Children's Health, University of New South Wales, Sydney, NSW, Australia.; Webster RI; Department of Neurology, Children's Hospital Westmead, Westmead, NSW, Australia.; Wenderoth JD; Department of Neurointervention, Prince of Wales Hospital, Randwick, NSW, Australia.; Andrews PI; Department of Neurology, Sydney Children's Hospital, Randwick, NSW, Australia.

Publisher: Blackwell Country of Publication: England NLM ID: 0006761 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1469-8749 (Electronic) Linking ISSN: 00121622 NLM ISO Abbreviation: Dev Med Child Neurol Subsets: MEDLINE

Di Donato N; Institute for Clinical Genetics, University Hospital, TU Dresden, 01307 Dresden, Germany.; Guerrini R; Pediatric Neurology, Neurogenetics and Neurobiology Unit and Laboratories, Meyer Children's Hospital, University of Florence, 50139 Florence, Italy.; Billington CJ; Department of Pediatrics, Division of Genetics and Metabolism, University of Minnesota, Minneapolis, MN 55454, USA.; Barkovich AJ; Departments of Radiology and Biomedical Imaging, Neurology, Pediatrics, and Neurosurgery, University of California, San Francisco, San Francisco, CA 94143, USA.; Dinkel P; Institute for Clinical Genetics, University Hospital, TU Dresden, 01307 Dresden, Germany.; Freri E; Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy.; Heide M; Max Planck Institute of Molecular Cell Biology and Genetics, 01307 Dresden, Germany.; German Primate Center, Leibniz Institute for Primate Research, 37077 Goettingen, Germany.; Gershon ES; Department of Human Genetics, The University of Chicago, Chicago, IL 60637, USA.; Department of Psychiatry and Behavioral Neuroscience, The University of Chicago, Chicago, IL 60637, USA.; Gertler TS; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA.; Hopkin RJ; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Department of Pediatrics, Division of Human Genetics, Cincinnati, OH 45229, USA.; Jacob S; Department of Psychiatry, University of Minnesota, Minneapolis, MN 55454, USA.; Keedy SK; Department of Psychiatry and Behavioral Neuroscience, The University of Chicago, Chicago, IL 60637, USA.; Kooshavar D; Bruce Lefory Centre, Murdoch Children's Research Institute and University of Melbourne Department of Pediatrics, Melbourne 3052, Australia.; Lockhart PJ; Bruce Lefory Centre, Murdoch Children's Research Institute and University of Melbourne Department of Pediatrics, Melbourne 3052, Australia.; Lohmann DR; Institut fur Humangenetik, Universitatsklinikum Essen, 45147 Essen, Germany.; Mahmoud IG; Pediatric Neurology Department, Cairo University Children's Hospital, Cairo, Egypt.; Parrini E; Pediatric Neurology, Neurogenetics and Neurobiology Unit and Laboratories, Meyer Children's Hospital, University of Florence, 50139 Florence, Italy.; Schrock E; Institute for Clinical Genetics, University Hospital, TU Dresden, 01307 Dresden, Germany.; Severi G; Medical Genetics Unit, S. Orsola-Malpighi Hospital, 40138 Bologna, Italy.; Timms AE; Center for Developmental Biology and Regenerative Medicine, Seattle Children's Research Institute, Seattle, WA 98101, USA.; Webster RI; T. Y. Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Sydney 2145, Australia.; Willis MJH; Uniformed Services University School of Medicine and Naval Medical Center, Department of Pediatrics, San Diego, CA 92134, USA.; Zaki MS; Pediatric Neurology Department, Cairo University Children's Hospital, Cairo, Egypt.; Clinical Genetics Department, Human Genetics and Genome Research Division, National Research Centre, Cairo Governorate 12622, Egypt.; Gleeson JG; Department of Neurosciences, Howard Hughes Medical Institute, University of California, San Diego, La Jolla, CA 92093, USA.; Leventer RJ; Department of Neurology, Royal Children's Hospital, Murdoch Children's Research Institute and University of Melbourne Department of Pediatrics, Melbourne 3052, Australia.; Dobyns WB; Department of Pediatrics, Division of Genetics and Metabolism, University of Minnesota, Minneapolis, MN 55454, USA.

Publisher: Oxford University Press Country of Publication: England NLM ID: 0372537 Publication Model: Print Cited Medium: Internet ISSN: 1460-2156 (Electronic) Linking ISSN: 00068950 NLM ISO Abbreviation: Brain Subsets: MEDLINE

Nicolas-Martinez EC; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; School of Biomedicine, University of Adelaide, Adelaide, SA 5005, Australia.; Robinson O; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; School of Biomedicine, University of Adelaide, Adelaide, SA 5005, Australia.; Pflueger C; Harry Perkins Institute of Medical Research, Nedlands, WA 6009, Australia; Australian Research Council Centre of Excellence in Plant Energy Biology, School of Molecular Sciences, The University of Western Australia, Crawley, WA 6009, Australia; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia.; Gardner A; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia.; Corbett MA; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia.; Ritchie T; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia.; Kroes T; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia.; van Eyk CL; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia.; Scheffer IE; Epilepsy Research Centre, Department of Medicine, The University of Melbourne, Austin Health, Heidelberg, VIC 3084, Australia; Murdoch Children's Research Institute, Parkville, VIC 3052, Australia; Florey Institute of Neuroscience and Mental Health, University of Melbourne, Parkville, VIC 3052, Australia; Department of Paediatrics, University of Melbourne, Royal Children's Hospital, Parkville, VIC 3052, Australia.; Hildebrand MS; Epilepsy Research Centre, Department of Medicine, The University of Melbourne, Austin Health, Heidelberg, VIC 3084, Australia; Department of Paediatrics, University of Melbourne, Royal Children's Hospital, Parkville, VIC 3052, Australia; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia.; Barnier JV; Institut des Neurosciences Paris-Saclay, UMR 9197, CNRS, Université Paris-Saclay, Saclay, France.; Rousseau V; Institut des Neurosciences Paris-Saclay, UMR 9197, CNRS, Université Paris-Saclay, Saclay, France.; Genevieve D; Montpellier University, Inserm U1183, Reference Center for Rare Diseases Developmental Anomaly and Malformative Syndromes, Genetics Department, Montpellier Hospital, Montpellier, France.; Haushalter V; Genetic Diagnosis Laboratory, Strasbourg University Hospital, Strasbourg, France.; Piton A; Genetic Diagnosis Laboratory, Strasbourg University Hospital, Strasbourg, France.; Denommé-Pichon AS; CRMRs 'Anomalies du Développement et syndromes malformatifs' et 'Déficiences Intellectuelles de causes rares', Centre de Génétique, CHU Dijon, Dijon, France; INSERM UMR1231, GAD 'Génétique des Anomalies du Développement,' FHU-TRANSLAD, University of Burgundy, Dijon, France.; Bruel AL; CRMRs 'Anomalies du Développement et syndromes malformatifs' et 'Déficiences Intellectuelles de causes rares', Centre de Génétique, CHU Dijon, Dijon, France; INSERM UMR1231, GAD 'Génétique des Anomalies du Développement,' FHU-TRANSLAD, University of Burgundy, Dijon, France.; Nambot S; CRMRs 'Anomalies du Développement et syndromes malformatifs' et 'Déficiences Intellectuelles de causes rares', Centre de Génétique, CHU Dijon, Dijon, France; INSERM UMR1231, GAD 'Génétique des Anomalies du Développement,' FHU-TRANSLAD, University of Burgundy, Dijon, France.; Isidor B; CRMRs 'Anomalies du Développement et syndromes malformatifs' et 'Déficiences Intellectuelles de causes rares', Centre de Génétique, CHU Dijon, Dijon, France; INSERM UMR1231, GAD 'Génétique des Anomalies du Développement,' FHU-TRANSLAD, University of Burgundy, Dijon, France.; Grigg J; Speciality of Ophthalmology, Save Sight Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2000, Australia.; Gonzalez T; Department of Clinical Genetics, Royal North Shore Hospital, St Leonards, NSW 2065, Australia.; Ghedia S; Department of Clinical Genetics, Royal North Shore Hospital, St Leonards, NSW 2065, Australia.; Marchant RG; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2000, Australia.; Bournazos A; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Children's Medical Research Institute, Westmead, NSW 2145, Australia.; Wong WK; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Children's Medical Research Institute, Westmead, NSW 2145, Australia; Department of Paediatric Neurology, Children's Hospital at Westmead, Sydney, NSW 2000, Australia.; Webster RI; Department of Paediatric Neurology, Children's Hospital at Westmead, Sydney, NSW 2000, Australia.; Evesson FJ; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2000, Australia; Children's Medical Research Institute, Westmead, NSW 2145, Australia.; Jones KJ; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Children's Medical Research Institute, Westmead, NSW 2145, Australia; Department of Clinical Genetics, Children's Hospital at Westmead, Sydney, NSW 2000, Australia.; Cooper ST; Kids Neuroscience Centre, Kids Research, Children's Hospital at Westmead, Westmead, NSW 2145, Australia; Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2000, Australia; Children's Medical Research Institute, Westmead, NSW 2145, Australia.; Lister R; Harry Perkins Institute of Medical Research, Nedlands, WA 6009, Australia; Australian Research Council Centre of Excellence in Plant Energy Biology, School of Molecular Sciences, The University of Western Australia, Crawley, WA 6009, Australia.; Gecz J; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; Adelaide Medical School, University of Adelaide, Adelaide, SA 5005, Australia; South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia. Electronic address: jozef.gecz@adelaide.edu.au.; Jolly LA; The Robinson Research Institute, University of Adelaide, Adelaide, SA 5005, Australia; School of Biomedicine, University of Adelaide, Adelaide, SA 5005, Australia. Electronic address: lachlan.jolly@adelaide.edu.au.

Publisher: Cell Press Country of Publication: United States NLM ID: 0370475 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1537-6605 (Electronic) Linking ISSN: 00029297 NLM ISO Abbreviation: Am J Hum Genet Subsets: MEDLINE

Dang UJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Damsker JM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Guglieri M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Clemens PR; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Perlman SJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Smith EC; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Horrocks I; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Finkel RS; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Mah JK; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Deconinck N; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Goemans NM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Haberlová J; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Straub V; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Mengle-Gaw L; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Schwartz BD; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Harper A; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Shieh PB; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; De Waele L; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Castro D; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Yang ML; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Ryan MM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; McDonald CM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Tulinius M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Webster RI; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Mcmillan HJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Kuntz N; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Rao VK; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Baranello G; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Spinty S; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Childs AM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Sbrocchi AM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Selby KA; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Monduy M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Nevo Y; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Vilchez JJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Nascimento-Osorio A; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Niks EH; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; De Groot IJM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Katsalouli M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Van Den Anker JN; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Ward LM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Leinonen M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; D'Alessandro AL; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.; Hoffman EP; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE

Alsharhan H; Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Department of Pediatrics, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait.; He M; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Edmondson AC; Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Daniel EJP; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Chen J; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Donald T; Pediatrics Ward, Grenada General Hospital, St. George's, Grenada.; Clinical Teaching Unit, St. George's University, St. George's, Grenada.; Bakhtiari S; Pediatric Movement Disorders Program, Division of Pediatric Neurology, Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, Arizona, USA.; Departments of Child Health, Neurology, Cellular & Molecular Medicine and Program in Genetics, University of Arizona College of Medicine, Phoenix, Arizona, USA.; Amor DJ; Murdoch Children's Research Institute, Royal Children's Hospital, Melbourne, and Department of Pediatrics, University of Melbourne, Melbourne, Australia.; Jones EA; Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester University NHS Foundation Trust, Manchester, UK.; Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.; Vassallo G; Department of Pediatric Neurology, Royal Manchester Children's Hospital, Manchester University Foundation Trust, Manchester, UK.; Vincent M; Service de génétique médicale, CHU de Nantes, Nantes, France.; Cogné B; Service de génétique médicale, CHU de Nantes, Nantes, France.; Deb W; Service de génétique médicale, CHU de Nantes, Nantes, France.; Werners AH; Department of Anatomy, Physiology and Pharmacology, St. George University School of Veterinary Medicine, St. George's, Grenada.; Jin SC; Department of Genetics and Pediatrics, Washington University, St. Louis, Missouri, USA.; Bilguvar K; Department of Genetics, Yale Center for Genome Analysis, Yale School of Medicine, New Haven, Connecticut, USA.; Christodoulou J; Brain and Mitochondrial Research Group, Murdoch Children's Research Institute, Royal Children's Hospital, Melbourne, and Department of Pediatrics, University of Melbourne, Melbourne, Australia.; Discipline of Child & Adolescent Health, Sydney Medical School, University of Sydney, Sydney, Australia.; Webster RI; Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Yearwood KR; St. George's University, University Health Services, St. George's, Grenada.; Ng BG; Human Genetics Program, Sanford Children's Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, California, USA.; Freeze HH; Human Genetics Program, Sanford Children's Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, California, USA.; Kruer MC; Pediatric Movement Disorders Program, Division of Pediatric Neurology, Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, Arizona, USA.; Departments of Child Health, Neurology, Cellular & Molecular Medicine and Program in Genetics, University of Arizona College of Medicine, Phoenix, Arizona, USA.; Li D; Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Raymond KM; Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA.; Bhoj EJ; Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Sobering AK; Department of Biochemistry, St. George's University School of Medicine, St. George's, Grenada.; Windward Islands Research and Education Foundation, True Blue, St. George's, Grenada.

Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE

Pelletier F; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Department of Specialized Medicine, Division of Medical Genetics, McGill University Health Centre, Montreal, QC, Canada.; Division of Child Neurology, Department of Pediatrics, CHU Sainte-Justine, Université de Montréal, Montreal, QC, Canada.; Perrier S; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Cayami FK; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, and Amsterdam Neuroscience, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.; Center of Biomedical Research, Faculty of Medicine, Diponegoro University, Semarang, Indonesia.; Mirchi A; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Department of Specialized Medicine, Division of Medical Genetics, McGill University Health Centre, Montreal, QC, Canada.; Saikali S; Department of Pathology, Centre Hospitalier Universitaire de Québec, Québec City, QC, Canada.; Tran LT; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Ulrick N; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Guerrero K; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Rampakakis E; Department of Pediatrics, McGill University, Montreal, QC, Canada.; van Spaendonk RML; Department of Clinical Genetics, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.; Naidu S; Department of Neurogenetics, Kennedy Krieger Institute, Johns Hopkins Medical Institutions, Baltimore, MD, USA.; Pohl D; Division of Neurology, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.; Gibson WT; Department of Medical Genetics, University of British Columbia, BC Children's Hospital Research Institute, Vancouver, BC, Canada.; Demos M; Division of Neurology, Department of Pediatrics, University of British Columbia, BC Children's Hospital, Vancouver, BC, Canada.; Goizet C; Centre de Référence Neurogénétique, Service de Génétique Médicale, Bordeaux University Hospital, and Laboratoire MRGM, INSERM U1211, Université de Bordeaux, Bordeaux, France.; Tejera-Martin I; Department of Neurology, Hospital Universitario Nuestra Señora de Candelaria, 38010 Santa Cruz de Tenerife, Canary Islands, Spain.; Potic A; Department of Neurology, Clinic for Child Neurology and Psychiatry, Medical Faculty University of Belgrade, Belgrade, Serbia.; Fogel BL; Departments of Neurology and Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.; Brais B; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Montreal Neurological Institute, Montreal, QC, Canada.; Sylvain M; Centre Mère Enfant, CHU de Québec, Québec City, QC, Canada.; Sébire G; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Pediatrics, Université de Sherbrooke, Sherbrooke, QC, Canada.; Lourenço CM; Faculdade de Medicina, Centro Universitario Estácio de Ribeirão Preto, Ribeirão Preto, SP, Brazil.; Bonkowsky JL; Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA.; Catsman-Berrevoets C; Department of Paediatric Neurology, Erasmus University Hospital - Sophia Children's Hospital, 3015 CN Rotterdam, The Netherlands.; Pinto PS; Neuroradiology Department, Centro Hospitalar do Porto, Porto, Portugal.; Tirupathi S; Department of Paediatric Neurology, Royal Belfast Hospital for Sick Children, Belfast, UK.; Strømme P; Division of Pediatrics and Adolescent Medicine, Oslo University Hospital, Ullevål, 0450 Oslo, and University of Oslo, Oslo, Norway.; de Grauw T; Department of Pediatrics, Emory School of Medicine, Atlanta, GA, USA.; Gieruszczak-Bialek D; Department of Medical Genetics, Children's Memorial Health Institute, Warsaw, Poland.; Department of Pediatrics, Medical University of Warsaw, Warsaw, Poland.; Krägeloh-Mann I; Department of Child Neurology, University Children's Hospital Tübingen, Tübingen, Germany.; Mierzewska H; Department of Child and Adolescent Neurology, Institute of Mother and Child, Warsaw, Poland.; Philippi H; Center of Developmental Neurology (SPZ Frankfurt Mitte), Frankfurt, Germany.; Rankin J; Peninsula Clinical Genetics Service, Royal Devon and Exeter NHS Foundation Trust, Exeter, UK.; Atik T; Division of Genetics, Department of Pediatrics, School of Medicine, Ege University, Izmir, Turkey.; Banwell B; Division of Neurology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Benko WS; Division of Pediatric Neurology, Department of Neurology, UC Davis Health System, Sacramento, CA, USA.; Blaschek A; Department of Pediatric Neurology and Developmental Medicine, Dr. v. Hauner Children's Hospital, University Hospital, LMU Munich, Munich, Germany.; Bley A; University Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Boltshauser E; Department of Child Neurology, University Children's Hospital Zurich, Zurich, Switzerland.; Bratkovic D; Metabolic Clinic, Women's and Children's Hospital, North Adelaide, South Australia, Australia.; Brozova K; Department of Child Neurology, Thomayers Hospital, Prague, Czech Republic.; Cimas I; Department of Neurology, Povisa Hospital, Vigo, Spain.; Clough C; Department of Neurology, King's College Hospital, London, UK.; Corenblum B; Division of Endocrinology & Metabolism, Department of Medicine, University of Calgary, Calgary, AB, Canada.; Dinopoulos A; Third Department of Pediatrics, National and Kapodistrian University of Athens, 'Attikon' Hospital, Athens, Greece.; Dolan G; Bristow Pediatrics, Bristow, VA, USA.; Faletra F; Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.; Fernandez R; Pediatric Neurology Associates, Tampa, FL, USA.; Fletcher J; Genetics and Molecular Pathology, Women's and Children's Hospital, Adelaide, South Australia, Australia.; Garcia Garcia ME; Department of Neurology, The Royal London Hospital, London, UK.; Gasparini P; Institute for Maternal and Child Health, IRCCS Burlo Garofolo, 34100 Trieste, and University of Trieste, Trieste, Italy.; Gburek-Augustat J; Division of Neuropaediatrics, Hospital for Children and Adolescents, University Leipzig, Leipzig, Germany.; Gonzalez Moron D; Neurogenetics Unit, Department of Neurology, Hospital JM Ramos Mejia, ADC, Buenos Aires, Argentina.; Hamati A; Department of Child Neurology, Indiana University, Indianapolis, IN, USA.; Harting I; Department of Neuroradiology, University Hospital Heidelberg, Heidelberg, Germany.; Hertzberg C; Department of Child Neurology, Vivantes Klinikum, Berlin, Germany.; Hill A; Department of Pediatrics, University of British Columbia, Vancouver, BC, Canada.; Hobson GM; Nemours Biomedical Research, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA.; Innes AM; Department of Medical Genetics and Alberta Children's Hospital Research Institute, University of Calgary, Calgary, AB, Canada.; Kauffman M; Neurogenetics Unit, Department of Neurology, Hospital JM Ramos Mejia and CONICET, ADC, Buenos Aires, Argentina.; Kirwin SM; Molecular Diagnostics Laboratory, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA.; Kluger G; PMU Salzburg, 5020 Salzburg, Austria; Clinic for Neuropediatrics and Neurorehabilitation, Epilepsy Center for Children and Adolescents, Schön Klinik Vogtareuth, Vogtareuth, Germany.; Kolditz P; Department of Child Neurology, Kantonsspital Luzern, Luzern, Switzerland.; Kotzaeridou U; Department of Child Neurology, University Children's Hospital Heidelberg, Heidelberg, Germany.; La Piana R; Department of Neuroradiology, Montreal Neurological Institute and Hospital, McGill University, Montreal, QC, Canada.; Liston E; Division of Clinical and Metabolic Genetics, The Hospital for Sick Children, Toronto, ON, Canada.; McClintock W; Pediatric Specialists of Virginia, Fairfax, VA, USA.; Department of Neurology, Children's National Medical Center, Washington, DC, USA.; McEntagart M; South West Thames Regional Genetics Service, St. George's Hospital, London, UK.; McKenzie F; Genetic Services of Western Australia, Subiaco, WA, Australia.; School of Paediatrics and Child Health, University of Western Australia, Perth, WA, Australia.; Melançon S; Department of Medical Genetics, McGill University Health Centre, Montreal Children's Hospital, Montreal, QC, Canada.; Misbahuddin A; Essex Centre for Neurological Sciences, Queen's Hospital, Romford, UK.; Suri M; Nottingham Clinical Genetics Service, City Hospital Campus, Nottingham University Hospitals NHS Trust, Nottingham, UK.; Monton FI; Department of Neurology, Hospital Universitario Nuestra Señora de Candelaria, 38010 Santa Cruz de Tenerife, Canary Islands, Spain.; Moutton S; Service de Génétique Médicale, CHU de Bordeaux, Bordeaux, France.; Murphy RPJ; Department of Neurology, Tallaght University Hospital, Tallaght, Ireland.; Nickel M; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Onay H; Department of Medical Genetics, Ege University, Izmir, Turkey.; Orcesi S; Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Pavia, Italy.; Özkınay F; Department of Pediatrics, Subdivision of Pediatric Genetics, Faculty of Medicine, Ege University, Izmir, Turkey.; Patzer S; Children's Hospital St. Elisabeth and St. Barbara, Halle (Saale), Germany.; Pedro H; Department of Pediatrics, The Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Hackensack, NJ, USA.; Pekic S; Clinic for Endocrinology, Diabetes and Diseases of Metabolism, University Clinical Center, Belgrade & School of Medicine, University of Belgrade, Belgrade, Serbia.; Pineda Marfa M; Hospital Sant Joan de Deu, Passeig de Sant Joan de Deu nº2, Barcelona, Spain.; Pizzino A; Department of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Genetics, MetroHealth Hospital, Cleveland, OH, USA.; Plecko B; Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University of Graz, Graz, Austria.; Poll-The BT; Department of Pediatric Neurology, Emma Children's Hospital, 1105 Amsterdam, The Netherlands.; Popovic V; Medical Faculty, University of Belgrade, Belgrade, Serbia.; Rating D; Department of Paediatric Neurology, University Children's Hospital, Heidelberg, Germany.; Rioux MF; Centre Hospitalier Universitaire de Sherbrooke - Hôpital Fleurimont, Sherbrooke, QC, Canada.; Rodriguez Espinosa N; Department of Neurology, Hospital Universitario Nuestra Señora de Candelaria, 38010 Santa Cruz de Tenerife, Canary Islands, Spain.; Ronan A; Hunter New England LHD, University of Newcastle, NSW, Australia.; Ostergaard JR; Centre for Rare Diseases, Aarhus University Hospital, Aarhus, Denmark.; Rossignol E; Departments of Neurosciences and Pediatrics, CHU-Sainte-Justine, Université de Montréal, Montreal, QC, Canada.; Sanchez-Carpintero R; Pediatric Neurology Unit, Department of Pediatrics, Clinica Universidad de Navarra, Pamplona, Spain.; Schossig A; Institute of Human Genetics, Medical University Innsbruck, Innsbruck, Austria.; Senbil N; Department of Child Neurology, Kırıkkale University Medical Faculty, Kırıkkale, Turkey.; Sønderberg Roos LK; Applied Human Molecular Genetics, Kennedy Center, Copenhagen University Hospital, Rigshospitalet, Glostrup, Denmark.; Stevens CA; Department of Pediatrics, Division of Medical Genetics, University of Tennessee College of Medicine, Chattanooga, TN, USA.; Synofzik M; Department of Neurodegeneration, Hertie Institute for Clinical Brain Research and Centre of Neurology, German Research Center for Neurodegenerative Diseases (DZNE), University of Tübingen, Tübingen, Germany.; Sztriha L; Department of Paediatrics, University of Szeged, Szeged, Hungary.; Tibussek D; Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital, Heinrich-Heine-University, 40225 Düsseldorf, Germany.; Timmann D; Department of Neurology, Essen University Hospital, University of Duisburg-Essen, Essen, Germany.; Tonduti D; Child Neurology Unit, V. Buzzi Children's Hospital, Milano, Italy.; van de Warrenburg BP; Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour, Radboud University Medical Centre, Nijmegen, The Netherlands.; Vázquez-López M; Sección Neuropediatría. Hospital Maternoinfantil Gregorio Marañón, Madrid, Spain.; Venkateswaran S; Division of Neurology, Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada.; Wasling P; Department of Neuroscience and Rehabilitation, The Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.; Wassmer E; Paediatric Neurology, Birmingham Children's Hospital, Birmingham, UK.; Webster RI; T. Y. Nelson Department of Neurology and Neurosurgery and the Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Wiegand G; Department of Pediatric Neurology, University Hospital Kiel, Germany.; Neuropediatrics Section of the Department of Pediatrics, Asklepios Clinic Hamburg Nord-Heidberg, Hamburg, Germany.; Yoon G; Division of Clinical and Metabolic Genetics, Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.; Rotteveel J; Emma Children's Hospital, Amsterdam UMC, Pediatric Endocrinology, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.; Schiffmann R; Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, TX, USA.; van der Knaap MS; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, and Amsterdam Neuroscience, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.; Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam, The Netherlands.; Vanderver A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.; Martos-Moreno GÁ; Department of Pediatric Endocrinology, Hospital Infantil Universitario Niño Jesús, Instituto de Investigación La Princesa, Madrid, Spain.; Department of Pediatrics, Universidad Autónoma de Madrid, 28049 Madrid, Spain.; CIBER de Fisiopatologia de la Obesidad y Nutriciόn (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain.; Polychronakos C; Division of Endocrinology, Montreal Children's Hospital and the Endocrine Genetics Lab, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Wolf NI; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, and Amsterdam Neuroscience, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.; Bernard G; Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada.; Department of Pediatrics, McGill University, Montreal, QC, Canada.; Department of Human Genetics, McGill University, Montreal, QC, Canada.; Child Health and Human Development Program, Research Institute of the McGill University Health Centre, Montreal, QC, Canada.; Department of Specialized Medicine, Division of Medical Genetics, McGill University Health Centre, Montreal, QC, Canada.

Publisher: Oxford University Press Country of Publication: United States NLM ID: 0375362 Publication Model: Print Cited Medium: Internet ISSN: 1945-7197 (Electronic) Linking ISSN: 0021972X NLM ISO Abbreviation: J Clin Endocrinol Metab Subsets: MEDLINE

Webster, Richard I; Majnemer, Annette; Platt, Robert W; Shevell, Michael I

Neurology; 2004 Dec 28, Vol. 63 Issue 12, p2327-2331, 5p

Oates EC; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; School of Biotechnology and Biomolecular Sciences, Faculty of Science, The University of New South Wales, Sydney, New South Wales, Australia.; Jones KJ; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Donkervoort S; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Charlton A; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Department of Histopathology, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Brammah S; Electron Microscope Unit, Department of Anatomical Pathology, Concord Repatriation General Hospital, Concord, Sydney, New South Wales, Australia.; Smith JE 3rd; Department of Cellular and Molecular Medicine, University of Arizona, Tucson, AZ.; Ware JS; National Heart and Lung Institute and MRC London Institute of Medical Science, Imperial College London, London, United Kingdom.; Royal Brompton and Harefield Hospitals NHS Trust, London, United Kingdom.; Yau KS; Institute for Medical Research and Centre for Medical Research, University of Western Australia, Nedlands, Western Australia, Australia.; Swanson LC; Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Whiffin N; National Heart and Lung Institute and MRC London Institute of Medical Science, Imperial College London, London, United Kingdom.; Royal Brompton and Harefield Hospitals NHS Trust, London, United Kingdom.; Peduto AJ; Department of Radiology, Westmead Hospital, Sydney, New South Wales, Australia.; University of Sydney Western Clinical School, Sydney, New South Wales, Australia.; Bournazos A; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Waddell LB; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Farrar MA; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia.; School of Women's and Children's Health, University of New South Wales Sydney, Sydney, New South Wales, Australia.; Sampaio HA; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia.; School of Women's and Children's Health, University of New South Wales Sydney, Sydney, New South Wales, Australia.; Teoh HL; Department of Neurology, Sydney Children's Hospital, Sydney, New South Wales, Australia.; School of Women's and Children's Health, University of New South Wales Sydney, Sydney, New South Wales, Australia.; Lamont PJ; Neurogenetic Unit, Department of Neurology, Royal Perth Hospital, Perth, Western Australia, Australia.; Mowat D; School of Women's and Children's Health, University of New South Wales Sydney, Sydney, New South Wales, Australia.; Department of Medical Genetics, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Fitzsimons RB; Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia.; Corbett AJ; Department of Neurology, Concord Repatriation General Hospital, Sydney, New South Wales, Australia.; Ryan MM; Department of Neurology, Royal Children's Hospital, Parkville, Victoria, Australia.; Murdoch Children's Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia.; University of Melbourne, Parkville, Victoria, Australia.; O'Grady GL; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Paediatric Neuroservices, Starship Child Health, Auckland, New Zealand.; Sandaradura SA; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Ghaoui R; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Joshi H; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Marshall JL; Analytic and Translational Genetics Unit, Massachusetts General Hospital, Boston, MA.; Medical and Population Genetics, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA.; Nolan MA; Paediatric Neuroservices, Starship Child Health, Auckland, New Zealand.; Kaur S; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Punetha J; Research Center for Genetic Medicine, Children's National Medical Center, Washington, DC.; Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, DC.; Töpf A; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Harris E; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Bakshi M; Department of Clinical Genetics, Liverpool Hospital, Sydney, New South Wales, Australia.; Genetti CA; Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Marttila M; Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Werlauff U; Danish National Rehabilitation Center for Neuromuscular Diseases, Aarhus, Denmark.; Streichenberger N; Neuropathology Department, Hospices Civils Lyon, Claude Bernard University, Lyon1, France.; NeuroMyogene Institute, CNRS UMR 5310, INSERM U1217, Lyon, France.; Pestronk A; Department of Neurology, Washington University School of Medicine, Saint Louis, MO.; Department of Pathology and Immunology, Washington University School of Medicine, Saint Louis, MO.; Mazanti I; Cellular Pathology, University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom.; Pinner JR; Department of Medical Genomics, Royal Prince Alfred Hospital, Camperdown, Sydney, New South Wales, Australia.; Vuillerot C; Woman-Mother-Child Hospital, Hospices Civils Lyon, Bron, France.; Claude Bernard University Lyon1, France.; Grosmann C; University of California, San Diego/Rady Children's Hospital, San Diego, CA.; Camacho A; Child Neurology Unit, Department of Neurology, October 12 University Hospital, Faculty of Medicine, Complutense University, Madrid, Spain.; Mohassel P; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Leach ME; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Bharucha-Goebel D; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Division of Neurology, Children's National Health System, Washington, DC.; Collins J; Mercy Clinic Pediatric Neurology, Springfield, MO.; Connolly AM; Neuromuscular Division, Departments of Neurology and Pediatrics, Washington University School of Medicine, Saint Louis, MO.; Gilbreath HR; Department of Advanced Practice, Children's Medical Center of Dallas, Dallas, TX.; Iannaccone ST; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX.; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX.; Castro D; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX.; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX.; Cummings BB; Analytic and Translational Genetics Unit, Massachusetts General Hospital, Boston, MA.; Broad Institute of Harvard and Massachusetts Institute of Technology, Cambridge, MA.; Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA.; Webster RI; T. Y. Nelson Department of Neurology and Neurosurgery, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Lazaro L; Pediatric Service, Basque Coast Hospital Center, Bayonne, France.; Vissing J; Neuromuscular Clinic and Research Unit, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.; Coppens S; Department of Pediatric Neurology, Neuromuscular Reference Center, Erasmus Hospital, Free University of Brussels, Brussels, Belgium.; Department of Pediatric Neurology, Neuromuscular Reference Center, Queen Fabiola Children's University Hospital, Free University of Brussels, Brussels, Belgium.; Deconinck N; Department of Pediatric Neurology, Neuromuscular Reference Center, Queen Fabiola Children's University Hospital, Free University of Brussels, Brussels, Belgium.; Luk HM; Clinical Genetic Service, Department of Health, Hong Kong, China.; Thomas NH; Department of Paediatric Neurology, University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom.; Foulds NC; Wessex Clinical Genetics Service, University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom.; Illingworth MA; Department of Paediatric Neurology, University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom.; Ellard S; University of Exeter Medical School, Exeter, United Kingdom.; Department of Molecular Genetics, Royal Devon and Exeter NHS Foundation Trust, Exeter, United Kingdom.; McLean CA; Department of Anatomical Pathology, Alfred Hospital, Melbourne, Victoria, Australia.; Faculty of Medicine, Nursing, and Health Sciences, Monash University, Melbourne, Victoria, Australia.; Phadke R; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.; National Hospital for Neurology and Neurosurgery, UCL Institute of Neurology, London, United Kingdom.; Ravenscroft G; Harry Perkins Institute, University of Western Australia, Nedlands, Western Australia, Australia.; Witting N; Copenhagen Neuromuscular Unit and Department of Neurology, Rigshospitalet, Copenhagen University, Copenhagen, Denmark.; Hackman P; Folkhälsan Institute of Genetics, Medicum, University of Helsinki, Helsinki, Finland.; Richard I; Généthon, INSERM U951, INTEGRARE Research Unit, Évry, France.; Cooper ST; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Kamsteeg EJ; Department of Human Genetics, Radboud University Medical Center, Nijmegen, the Netherlands.; Hoffman EP; Research Center for Genetic Medicine, Children's National Medical Center, Washington, DC.; Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, DC.; Bushby K; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Straub V; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Udd B; Folkhälsan Institute of Genetics, Medicum, University of Helsinki, Helsinki, Finland.; Neuromuscular Research Center, Tampere University and University Hospital, Neurology, Tampere, Finland.; Department of Medical Genetics, University of Helsinki, Helsinki, Finland.; Vaasa Central Hospital, Department of Neurology, Vaasa, Finland.; Ferreiro A; Pathophysiology of Striated Muscles Laboratory, Unit of Functional and Adaptative Biology, BFA, Paris Diderot University/CNRS, Sorbonne Paris Cité, Paris, France.; Public Hospital Network of Paris, Paris-East Reference Center Neuromuscular Diseases, Pitié-Salpêtrière Hospital Group, Paris, France.; North KN; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Murdoch Children's Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia.; Clarke NF; Institute for Neuroscience and Muscle Research, Kid's Research Institute, Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.; Lek M; Analytic and Translational Genetics Unit, Massachusetts General Hospital, Boston, MA.; Broad Institute of Harvard and Massachusetts Institute of Technology, Cambridge, MA.; Beggs AH; Manton Center for Orphan Disease Research, Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.; MacArthur DG; Analytic and Translational Genetics Unit, Massachusetts General Hospital, Boston, MA.; Broad Institute of Harvard and Massachusetts Institute of Technology, Cambridge, MA.; Granzier H; Department of Cellular and Molecular Medicine, University of Arizona, Tucson, AZ.; Davis MR; Department of Diagnostic Genomics, PathWest Laboratory Medicine WA, Nedlands, Western Australia, Australia.; Laing NG; Harry Perkins Institute, University of Western Australia, Nedlands, Western Australia, Australia.

Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann Neurol

Richard EM; Department of Otorhinolaryngology Head and Neck Surgery, School of Medicine, University of Maryland, Baltimore, MD 21201, USA.; Bakhtiari S; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Marsh APL; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Kaiyrzhanov R; Department of Neuromuscular Disorders, Institute of Neurology, University College London, Queen Square, WC1N 3BG London, UK.; Wagner M; Institute of Human Genetics, Klinikum rechts der Isar, School of Medicine, Technical University of Munich, 81675 Munich, Germany; Institute of Neurogenomics, Helmholtz Zentrum München, 85764 Neuherberg, Germany.; Shetty S; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Pagnozzi A; CSIRO Health and Biosecurity, The Australian e-Health Research Centre, Brisbane, QLD 4029, Australia.; Nordlie SM; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Guida BS; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Cornejo P; Pediatric Neuroradiology Division, Pediatric Radiology, Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; University of Arizona College of Medicine, Phoenix, AZ 85004, USA; Mayo Clinic, Scottsdale, AZ 85259, USA.; Magee H; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Liu J; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Norton BY; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Webster RI; Neurology Department, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.; Worgan L; Department of Medical Genomics, Royal Prince Alfred Hospital, Sydney, NSW 2050, Australia.; Hakonarson H; Center for Applied Genomics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Li J; Department of Computer Science, City University of Hong Kong, Kowloon 999077, Hong Kong.; Guo Y; Center for Applied Genomics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19146, USA.; Jain M; Department of Bone and Osteogenesis Imperfecta, Kennedy Krieger Institute, Baltimore, MD 21205, USA.; Blesson A; Center for Autism and Related Disorders, Kennedy Krieger Institute, Baltimore, MD 21211, USA.; Rodan LH; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115, USA; Department of Neurology, Boston Children's Hospital, Boston, MA 02115, USA.; Abbott MA; University of Massachusetts Medical School - Baystate, Baystate Children's Hospital, Springfield, MA 01107, USA.; Comi A; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA.; Cohen JS; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA.; Alhaddad B; Institute of Human Genetics, Klinikum rechts der Isar, School of Medicine, Technical University of Munich, 81675 Munich, Germany.; Meitinger T; Institute of Human Genetics, Klinikum rechts der Isar, School of Medicine, Technical University of Munich, 81675 Munich, Germany.; Lenz D; Centre of Child and Adolescent Medicine, Department of Pediatric Neurology and Metabolic Medicine, Heidelberg University Hospital, 69120 Heidelberg, Germany.; Ziegler A; Department of Child Neurology and Metabolic Medicine, Center for Pediatric and Adolescent Medicine, University Hospital Heidelberg, Im Neuenheimer Feld 430, 69120 Heidelberg, Germany.; Kotzaeridou U; Department of Child Neurology and Metabolic Medicine, Center for Pediatric and Adolescent Medicine, University Hospital Heidelberg, Im Neuenheimer Feld 430, 69120 Heidelberg, Germany.; Brunet T; Institute of Human Genetics, Klinikum rechts der Isar, School of Medicine, Technical University of Munich, 81675 Munich, Germany.; Chassevent A; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, MD 21205, USA.; Smith-Hicks C; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, MD 21205, USA; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA.; Ekstein J; Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, New York, NY 11211, USA.; Weiden T; Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Jerusalem 9054020, Israel.; Hahn A; Department of Child Neurology, Justus-Liebig-University Giessen, 35392 Giessen, Germany.; Zharkinbekova N; Department of Neurology, South Kazakhstan Medical Academy, Shymkent 160001, Kazakhstan.; Turnpenny P; Clinical Genetics, Royal Devon & Exeter NHS Foundation Trust, EX1 2ED Exeter, UK.; Tucci A; Clinical Pharmacology, William Harvey Research Institute, Charterhouse Square, School of Medicine and Dentistry Queen Mary University of London, London EC1M 6BQ, UK.; Yelton M; Penn State Health Children's Hospital, Hershey, PA 17033, USA.; Horvath R; Department of Clinical Neurosciences, John Van Geest Cambridge Centre for Brain Repair, University of Cambridge School of Clinical Medicine, CB2 0PY Cambridge, UK.; Gungor S; Inonu University, Faculty of Medicine, Turgut Ozal Research Center, Department of Paediatric Neurology, 44280 Malatya, Turkey.; Hiz S; Izmir Biomedicine and Genome Center, Dokuz Eylul University Health Campus, 35340 Izmir, Turkey; Department of Pediatric Neurology, Faculty of Medicine, Dokuz Eylul University, 35340 Izmir, Turkey.; Oktay Y; Izmir Biomedicine and Genome Center, Dokuz Eylul University Health Campus, 35340 Izmir, Turkey; Department of Medical Biology, Faculty of Medicine, Dokuz Eylul University, 35220 Izmir, Turkey.; Lochmuller H; Children's Hospital of Eastern Ontario Research Institute; Division of Neurology, Department of Medicine, The Ottawa Hospital, and Brain and Mind Research Institute, University of Ottawa, Ottawa, ON K1H 8L1, Canada.; Zollino M; Università Cattolica Sacro Cuore, Facoltà di Medicina e Chirurgia, Dipartimento Scienze della Vita e Sanità Pubblica, 00168 Roma, Italy; Fondazione Policlinico A. Gemelli IRCCS, Sezione di Medicina Genomica, 00168 Roma, Italy.; Morleo M; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Naples, Italy.; Marangi G; Università Cattolica Sacro Cuore, Facoltà di Medicina e Chirurgia, Dipartimento Scienze della Vita e Sanità Pubblica, 00168 Roma, Italy; Fondazione Policlinico A. Gemelli IRCCS, Sezione di Medicina Genomica, 00168 Roma, Italy.; Nigro V; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Naples, Italy; Department of Precision Medicine, University of Campania 'Luigi Vanvitelli,' 80138 Naples, Italy.; Torella A; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Naples, Italy; Department of Precision Medicine, University of Campania 'Luigi Vanvitelli,' 80138 Naples, Italy.; Pinelli M; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Naples, Italy.; Amenta S; Università Cattolica Sacro Cuore, Facoltà di Medicina e Chirurgia, Dipartimento Scienze della Vita e Sanità Pubblica, 00168 Roma, Italy; Fondazione Policlinico A. Gemelli IRCCS, Sezione di Medicina Genomica, 00168 Roma, Italy.; Husain RA; Department of Neuropediatrics, Jena University Hospital, 07747 Jena, Germany.; Grossmann B; Institute of Medical Genetics and Applied Genomics, University of Tübingen, 72076 Tuebingen, Germany.; Rapp M; Institute of Systems Motor Science, University of Lübeck, 23538 Lübeck, Germany.; Steen C; Department of Paediatric and Adolescent Medicine, St Joseph Hospital, 12101 Berlin, Germany.; Marquardt I; University Children's Hospital Oldenburg, Department of Neuropaediatric and Metabolic Diseases, 26133 Oldenburg, Germany.; Grimmel M; Institute of Medical Genetics and Applied Genomics, University of Tübingen, 72076 Tuebingen, Germany.; Grasshoff U; Institute of Medical Genetics and Applied Genomics, University of Tübingen, 72076 Tuebingen, Germany.; Korenke GC; University Children's Hospital Oldenburg, Department of Neuropaediatric and Metabolic Diseases, 26133 Oldenburg, Germany.; Owczarek-Lipska M; Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany; Junior Research Group, Genetics of Childhood Brain Malformations, Faculty VI-School of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany.; Neidhardt J; Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany; Research Center Neurosensory Science, University of Oldenburg, 26129 Oldenburg, Germany.; Radio FC; Genetics and Rare Diseases Research Division, Ospedale Pediatrico Bambino Gesù, IRCCS, 00146 Rome, Italy.; Mancini C; Genetics and Rare Diseases Research Division, Ospedale Pediatrico Bambino Gesù, IRCCS, 00146 Rome, Italy.; Claps Sepulveda DJ; Genetics and Rare Diseases Research Division, Ospedale Pediatrico Bambino Gesù, IRCCS, 00146 Rome, Italy.; McWalter K; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Begtrup A; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Crunk A; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Guillen Sacoto MJ; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Person R; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Schnur RE; GeneDx, 207 Perry Parkway, Gaithersburg, MD 20877, USA.; Mancardi MM; Unit of Child Neuropsichiatry, Department of Clinical and Surgical Neurosciences and Rehabilitation, IRCCS Giannina Gaslini, Genoa 16147, Italy.; Kreuder F; Australian Regenerative Medicine Institute, Monash University, Clayton, VIC 3168, Australia.; Striano P; Pediatric Neurology and Muscular Diseases Unit, IRRCS Istituto Giannina Gaslini, 16148 Genoa, Italy; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, 16142 Genoa, Italy.; Zara F; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, 16142 Genoa, Italy; Unit of Medical Genetics, IRRCS Istituto Giannina Gaslini, 16147 Genoa, Italy.; Chung WK; Departments of Pediatrics and Medicine, Columbia University, New York, NY 10032, USA.; Marks WA; Department of Neurology, Cook Children's Medical Center, Fort Worth, TX 76104, USA; Department of Pediatrics, University of North Texas Health Science Center, Fort Worth, TX 76107, USA.; van Eyk CL; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; Webber DL; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; Corbett MA; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; Harper K; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; Berry JG; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; MacLennan AH; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia.; Gecz J; Robinson Research Institute, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5006, Australia; Adelaide Medical School, Faculty of Health and Medical Sciences, University of Adelaide, Adelaide, SA 5000, Australia; South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia.; Tartaglia M; Genetics and Rare Diseases Research Division, Ospedale Pediatrico Bambino Gesù, IRCCS, 00146 Rome, Italy.; Salpietro V; Pediatric Neurology and Muscular Diseases Unit, IRRCS Istituto Giannina Gaslini, 16148 Genoa, Italy; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, 16142 Genoa, Italy.; Christodoulou J; Brain and Mitochondrial Research Group, Murdoch Children's Research Institute, Melbourne Department of Paediatrics, University of Melbourne, Melbourne, VIC 3052, Australia; Discipline of Child and Adolescent Health, University of Sydney, Sydney, NSW 2006, Australia.; Kaslin J; Australian Regenerative Medicine Institute, Monash University, Clayton, VIC 3168, Australia.; Padilla-Lopez S; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA.; Bilguvar K; Yale Center for Genome Analysis, Yale University, New Haven, CT 06520, USA; Department of Genetics, Yale University School of Medicine, New Haven, CT 06510, USA.; Munchau A; Institute of Systems Motor Science, University of Lübeck, 23538 Lübeck, Germany.; Ahmed ZM; Department of Otorhinolaryngology Head and Neck Surgery, School of Medicine, University of Maryland, Baltimore, MD 21201, USA; Department of Biochemistry and Molecular Biology, School of Medicine, University of Maryland, Baltimore, MD 21201, USA.; Hufnagel RB; Ophthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA.; Fahey MC; Department of Paediatrics, Monash University, Melbourne, VIC 3168, Australia.; Maroofian R; Department of Neuromuscular Disorders, Institute of Neurology, University College London, Queen Square, WC1N 3BG London, UK.; Houlden H; Department of Neuromuscular Disorders, Institute of Neurology, University College London, Queen Square, WC1N 3BG London, UK.; Sticht H; Institute of Biochemistry, Friedrich-Alexander-Universität Erlangen-Nürnberg, 91054 Erlangen, Germany.; Mane SM; Yale Center for Genome Analysis, Yale University, New Haven, CT 06520, USA; Department of Genetics, Yale University School of Medicine, New Haven, CT 06510, USA.; Rad A; Department of Otolaryngology - Head and Neck Surgery, Tübingen Hearing Research Centre, Eberhard Karls University Tübingen, 72076 Tübingen, Germany.; Vona B; Department of Otolaryngology - Head and Neck Surgery, Tübingen Hearing Research Centre, Eberhard Karls University Tübingen, 72076 Tübingen, Germany.; Jin SC; Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110, USA.; Haack TB; Institute of Medical Genetics and Applied Genomics, University of Tübingen, 72076 Tuebingen, Germany; Centre for Rare Diseases, University of Tübingen, 72074 Tuebingen, Germany.; Makowski C; Department of Paediatrics, Adolescent Medicine and Neonatology, Munich Clinic, Schwabing Hospital and Technical University of Munich, School of Medicine, 80804 Munich, Germany.; Hirsch Y; Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, New York, NY 11211, USA.; Riazuddin S; Department of Otorhinolaryngology Head and Neck Surgery, School of Medicine, University of Maryland, Baltimore, MD 21201, USA; Department of Biochemistry and Molecular Biology, School of Medicine, University of Maryland, Baltimore, MD 21201, USA. Electronic address: sriazuddin@som.umaryland.edu.; Kruer MC; Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, AZ 85016, USA; Departments of Child Health, Neurology, Cellular, and Molecular Medicine and Program in Genetics, University of Arizona College of Medicine - Phoenix, Phoenix, AZ 85004, USA. Electronic address: kruerm@arizona.edu.

Publisher: Cell Press Country of Publication: United States NLM ID: 0370475 Publication Model: Print Cited Medium: Internet ISSN: 1537-6605 (Electronic) Linking ISSN: 00029297 NLM ISO Abbreviation: Am J Hum Genet Subsets: MEDLINE

Thiffault I; 1] Department of Neurology and Neurosurgery, McGill University, Department of Medical Genetics, Montreal Children's Hospital, Research Institute of the McGill University Health Center, 1001 boul Décarie, Montreal, Quebec H4A 3J1, Canada. [2] Service de Génétique, Centre Hospitalier Universitaire Sainte-Justine, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, Quebec H3T1C5, Canada. [3] Center for Pediatric Genomic Medicine, Children's Mercy Hospital, 2420 Pershing Road, Suite 421, Kansas City, Missouri 64108, USA.; Wolf NI; Department of Child Neurology, VU University Medical Center, Neuroscience Campus Amsterdam, Amsterdam 1081 HZ, The Netherlands.; Forget D; Translational Proteomics Laboratory, Institut de recherches cliniques de Montréal (IRCM), 110 avenue des Pins ouest, Montréal, Québec H2W 1R7, Canada.; Guerrero K; Department of Neurology and Neurosurgery, McGill University, Department of Medical Genetics, Montreal Children's Hospital, Research Institute of the McGill University Health Center, 1001 boul Décarie, Montreal, Quebec H4A 3J1, Canada.; Tran LT; Department of Neurology and Neurosurgery, McGill University, Department of Medical Genetics, Montreal Children's Hospital, Research Institute of the McGill University Health Center, 1001 boul Décarie, Montreal, Quebec H4A 3J1, Canada.; Choquet K; Neurogenetics of Motion Laboratory, Montreal Neurological Institute, 3801 University Street, McGill University, Montreal, Quebec H3A 2B4, Canada.; Lavallée-Adam M; Department of Chemical Physiology, The Scripps Research Institute, 10550 North Torrey Pines Road SR302, La Jolla, California 92037, USA.; Poitras C; Translational Proteomics Laboratory, Institut de recherches cliniques de Montréal (IRCM), 110 avenue des Pins ouest, Montréal, Québec H2W 1R7, Canada.; Brais B; Neurogenetics of Motion Laboratory, Montreal Neurological Institute, 3801 University Street, McGill University, Montreal, Quebec H3A 2B4, Canada.; Yoon G; Division of Neurology and Clinical and Metabolic Genetics, the Hospital for Sick Children, University of Toronto, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada.; Sztriha L; Department of Paediatrics, Faculty of Medicine, University of Szeged, Temesvári krt. 35-37, Szeged H-6726, Hungary.; Webster RI; 1] T.Y. Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Locked Bag 4001, Westmead, New South Wales 2145, Australia. [2] Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead, Locked Bag 4001, Westmead New South Wales 2145, Australia.; Timmann D; Department of Neurology, University Clinic Essen, University of Duisburg-Essen, Hufelandstrasse 55, 45147 Essen, Germany.; van de Warrenburg BP; Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour, Radboud University Medical Center, PO Box 9101, Nijmegen 6500 HB, The Netherlands.; Seeger J; Department of Pediatrics and Adolescent Medicine, Deutsche KlinikfürDiagnostik, Wiesbaden 65191, Germany.; Zimmermann A; Department of Paediatrics, Faculty of Medicine, University of Szeged, Temesvári krt. 35-37, Szeged H-6726, Hungary.; Máté A; Department of Neurosurgery, Faculty of Medicine, University of Szeged, 6 Semmelweis Street, Szeged H-6725, Hungary.; Goizet C; Service de Génétique, Hôpital Pellegrin, CHU Bordeaux and University Bordeaux, Laboratoire MRGM (EA4576), Bordeaux 33076, France.; Fung E; Department of Paediatrics, The Chinese University of Hong Kong, Prince of Wales Hospital, Shatin, Hong Kong, SAR China.; van der Knaap MS; Department of Child Neurology, VU University Medical Center, Neuroscience Campus Amsterdam, Amsterdam 1081 HZ, The Netherlands.; Fribourg S; 1] Université de Bordeaux, Institut Européen de Chimie et Biologie, ARNA Laboratory, Pessac F-33607, France. [2] Institut National de la Santé Et de la Recherche Médicale, INSERM-U869, ARNA Laboratory, Bordeaux F-33000, France.; Vanderver A; 1] Center for Genetic Medicine Research, Children's National, 111 Michigan Avenue Northwest, Washington, District of Columbia 20010, USA. [2] Department of Neurology, Children's National, 111 Michigan Avenue Northwest, Washington, District of Columbia 20010, USA. [3] George Washington University, School of Medicine, Washington, District of Columbia 20052, USA.; Simons C; Institute for Molecular Bioscience, University of Queensland, Brisbane, Queensland 4072, Australia.; Taft RJ; 1] George Washington University, School of Medicine, Washington, District of Columbia 20052, USA. [2] Institute for Molecular Bioscience, University of Queensland, Brisbane, Queensland 4072, Australia. [3] Departments of Integrative Systems Biology and Pediatrics, School of Medicine and Health Sciences, The George Washington University, Washington, District of Columbia 20037, USA. [4] Illumina Inc., 5200 Illumina Way, San Diego, California 92122, USA.; Yates JR 3rd; Department of Chemical Physiology, The Scripps Research Institute, 10550 North Torrey Pines Road SR302, La Jolla, California 92037, USA.; Coulombe B; 1] Translational Proteomics Laboratory, Institut de recherches cliniques de Montréal (IRCM), 110 avenue des Pins ouest, Montréal, Québec H2W 1R7, Canada. [2] Department of Biochemistry, Université de Montréal, Pavillon Roger-Gaudry, CP 6128, Succ Centre-Ville, Montreal, Québec H3C 3J7, Canada.; Bernard G; Department of Neurology and Neurosurgery, McGill University, Department of Medical Genetics, Montreal Children's Hospital, Research Institute of the McGill University Health Center, 1001 boul Décarie, Montreal, Quebec H4A 3J1, Canada.

Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

Mohammad SS; Kids Neuroscience Centre, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.; TY Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Sydney, Australia.; The Children's hospital at Westmead Clinical School, Faculty of Medicine, University of Sydney, Sydney, NSW 2145, Australia.; Angiti RR; Newborn and Peadiatric Emergency Transport Service (NETS), Bankstown, NSW, Australia.; Department of Neonatology, Liverpool Hospital, Liverpool, NSW, Australia.; Biggin A; The Children's hospital at Westmead Clinical School, Faculty of Medicine, University of Sydney, Sydney, NSW 2145, Australia.; Morales-Briceño H; Movement Disorders Unit, Neurology Department, Westmead Hospital, Westmead, NSW 2145, Australia.; Goetti R; Medical Imaging, The Children's Hospital at Westmead and Sydney Medical School, University of Sydney, Sydney, Australia.; Perez-Dueñas B; Paediatric Neurology Department, Hospital Vall d'Hebrón Universitat Autónoma de Barcelona, Vall d'Hebron Research Institute Barcelona, Barcelona, Spain.; Gregory A; Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA.; Hogarth P; Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA.; Ng J; Molecular Neurosciences, Developmental Neurosciences, UCL-Institute of Child Health, London, UK.; Papandreou A; Molecular Neurosciences, Developmental Neurosciences, UCL-Institute of Child Health, London, UK.; Bhattacharya K; Western Sydney Genomics Program, The Children's Hospital at Westmead and Sydney Medical School, University of Sydney, Sydney, Australia.; Rahman S; Mitochondrial Research Group, Genetics and Genomic Medicine, Institute of Child Health, University College London and Metabolic Unit, Great Ormond Street Hospital, London, UK.; Prelog K; Medical Imaging, The Children's Hospital at Westmead and Sydney Medical School, University of Sydney, Sydney, Australia.; Webster RI; TY Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Sydney, Australia.; Wassmer E; Department of Paediatric Neurology, Birmingham Children's Hospital, Birmingham, UK.; Hayflick S; Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA.; Livingston J; Department of Paediatric Neurology, Leeds Teaching Hospitals Trust, University of Leeds, UK.; Kurian M; Molecular Neurosciences, Developmental Neurosciences, UCL-Institute of Child Health, London, UK.; Chong WK; Department of Radiology, Great Ormond Street Hospital, London, UK.; Dale RC; Kids Neuroscience Centre, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.; TY Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Sydney, Australia.; The Children's hospital at Westmead Clinical School, Faculty of Medicine, University of Sydney, Sydney, NSW 2145, Australia.

Publisher: Oxford University Press Country of Publication: England NLM ID: 101755125 Publication Model: eCollection Cited Medium: Internet ISSN: 2632-1297 (Electronic) Linking ISSN: 26321297 NLM ISO Abbreviation: Brain Commun Subsets: PubMed not MEDLINE

Scrivener TA; Faculty of Medicine, University of Sydney, Sydney, NSW, Australia. tscr9663@uni.sydney.edu.au.; Ross SM; Street NE; Webster RI; De Lima JC

Publisher: Arnette-Blackwell Country of Publication: France NLM ID: 9206575 Publication Model: Print Cited Medium: Internet ISSN: 1460-9592 (Electronic) Linking ISSN: 11555645 NLM ISO Abbreviation: Paediatr Anaesth Subsets: MEDLINE

Webster RI; Department of Neurology/Neurosurgery, McGill University Montreal, Children's Hospital Education Research Institute, Children's Hospital at Westmead, Westmead, NSW, Australia.; Erdos C; Evans K; Majnemer A; Saigal G; Kehayia E; Thordardottir E; Evans A; Shevell MI

Publisher: Sage Country of Publication: United States NLM ID: 8606714 Publication Model: Print Cited Medium: Internet ISSN: 1708-8283 (Electronic) Linking ISSN: 08830738 NLM ISO Abbreviation: J Child Neurol Subsets: MEDLINE

Young HK; Institute for Neuromuscular Research, The Children's Hospital at Westmead, Sydney, Australia. hyoung@nsccahs.health.nsw.gov.au; Barton BA; Waisbren S; Portales Dale L; Ryan MM; Webster RI; North KN

Publisher: Sage Country of Publication: United States NLM ID: 8606714 Publication Model: Print-Electronic Cited Medium: Print ISSN: 0883-0738 (Print) Linking ISSN: 08830738 NLM ISO Abbreviation: J Child Neurol Subsets: MEDLINE

Webster RI; Department of Neurology, School of Pysical and Occupational Therapy, McGill University, Montreal, Quebec, Canada.; Majnemer A; Platt RW; Shevell MI

Publisher: Sage Country of Publication: United States NLM ID: 8606714 Publication Model: Print Cited Medium: Print ISSN: 0883-0738 (Print) Linking ISSN: 08830738 NLM ISO Abbreviation: J Child Neurol Subsets: MEDLINE

Oskoui M; Department of Neurology/Neurosurgery McGill University, Division of Pediatric Neurology, Montreal Children's Hospital, Montreal, QC, Canada.; Webster RI; Zhang X; Shevell MI

Publisher: Sage Country of Publication: United States NLM ID: 8606714 Publication Model: Print Cited Medium: Print ISSN: 0883-0738 (Print) Linking ISSN: 08830738 NLM ISO Abbreviation: J Child Neurol Subsets: MEDLINE

Webster RI; Department of Neurology, McGill University, Montreal, QC, Canada. richard.webster@muhc.mcgill.ca; Shevell MI

Publisher: Sage Country of Publication: United States NLM ID: 8606714 Publication Model: Print Cited Medium: Print ISSN: 0883-0738 (Print) Linking ISSN: 08830738 NLM ISO Abbreviation: J Child Neurol Subsets: MEDLINE

Vlaskamp DRM; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Shaw BJ; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Burgess R; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Mei D; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Montomoli M; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Xie H; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Myers CT; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Bennett MF; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; XiangWei W; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Williams D; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Maas SM; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Brooks AS; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Mancini GMS; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; van de Laar IMBH; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; van Hagen JM; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Ware TL; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Webster RI; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Malone S; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Berkovic SF; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Kalnins RM; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Sicca F; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Korenke GC; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; van Ravenswaaij-Arts CMA; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Hildebrand MS; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Mefford HC; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Jiang Y; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Guerrini R; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia.; Scheffer IE; From the Epilepsy Research Centre (D.R.M.V., B.J.S., R.B., M.F.B., S.F.B., M.S.H., I.E.S.), Department of Medicine, University of Melbourne, Austin Health, Australia; Departments of Genetics (D.R.M.V., C.M.A.v.R.-A.) and Neurology (D.R.M.V.), University Medical Center Groningen, University of Groningen, the Netherlands; Pediatric Neurology Unit and Laboratories (D.M., M.M.) and Pediatric Neurology (R.G.), Neurogenetics and Neurobiology Unit and Laboratories, A. Meyer Children's Hospital, University of Florence, Italy; Department of Pediatrics and Pediatric Epilepsy Centre (H.X., W.X.W., Y.J.), Peking University First Hospital, Beijing, China; Department of Pediatrics (C.T.M., H.C.M.), Division of Genetic Medicine, University of Washington, Seattle; Population Health and Immunity Division (M.F.B.), Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia; Department of Medical Biology (M.F.B.), University of Melbourne, Australia; Caulfield (D.W.), Melbourne, Australia; Department of Clinical Genetics (S.M.M.), Academic Medical Centre, Amsterdam, the Netherlands; Department of Clinical Genetics (A.S.B., G.M.S.M., I.M.B.H.v.d.L.), Erasmus University Medical Centre, Rotterdam, the Netherlands; Department of Clinical Genetics (J.M.v.H.), VU University Medical Center, Amsterdam, the Netherlands; Tasmanian Health Service (T.L.W.), Women's and Children's Services, Launceston General Hospital, Tasmania, Australia; TY Nelson Department of Neurology and Neurosurgery (R.I.W.) and Institute of Neuroscience and Muscle Research (R.I.W.), Children's Hospital at Westmead, Sydney, Australia; Department of Neurosciences (S.M.), Lady Cilento Children's Hospital, Brisbane, Australia; Department of Anatomical Pathology (R.M.K.), Austin Hospital, Melbourne, Australia; IRCCS Stella Maris Foundation (F.S., R.G.), Pisa, Italy; Klinikum Oldenburg (G.C.K.), Zentrum für Kinder-und Jugendmedizin, Klinik für Neuropädiatrie u. angeborene Stoffwechselerkrankungen, Oldenburg, Germany; Centre of Epilepsy (Y.J.), Beijing Institute for Brain Disorders, China; Department of Paediatrics (I.E.S.), University of Melbourne, Royal Children's Hospital, Australia; and Florey Institute of Neurosciences and Mental Health (I.E.S.), Parkville, Australia. scheffer@unimelb.edu.au.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE