부산대학교 도서관

Finch M; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; McGaw Medical Center of Northwestern University, Chicago, IL, USA.; Oswald S; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; McGaw Medical Center of Northwestern University, Chicago, IL, USA.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; Schwaede A; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

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

Tannu, Nilesh S ; Wu, Jian ; Rao, Vamshi K ; Gadgil, Himanshu S ; Pabst, Michael J ; Gerling, Ivan C ; Raghow, Rajendra

In Analytical Biochemistry 2004 327(2):222-232

Toaz EE; Department of Pediatric Anesthesiology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Pinto NM; Department of Pediatric Anesthesiology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Rao VK; Division of Neurology, Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Cheon EC; Department of Pediatric Anesthesiology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 7503323 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2125 (Electronic) Linking ISSN: 03065251 NLM ISO Abbreviation: Br J Clin Pharmacol Subsets: MEDLINE

Drackley A; Department of Pathology & Laboratory Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Division of Genetics, Genomics, and Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; De Simone L; Division of Genetics, Genomics, and Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Division of Neurology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Kuntz N; Division of Neurology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Rahmani S; Division of Ophthalmology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Ophthalmology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Ing A; Department of Pathology & Laboratory Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Division of Genetics, Genomics, and Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Rao VK; Division of Neurology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Rathbun P; Department of Pathology & Laboratory Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Pathology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Yap KL; Department of Pathology & Laboratory Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.; Department of Pathology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 101235741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1552-4833 (Electronic) Linking ISSN: 15524825 NLM ISO Abbreviation: Am J Med Genet A Subsets: MEDLINE

Munson HE; Chicago College of Osteopathic Medicine, Midwestern University, Downers Grove, IL, USA. hannahmunson@gmail.com.; De Simone L; Division of Genetics, Birth Defects and Metabolism, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Schwaede A; Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Bhatia A; Department of Speech-Language Pathology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Mithal DS; Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Stanley Manne Children's Research Institute, Chicago, IL, USA.; Young N; Stanley Manne Children's Research Institute, Chicago, IL, USA.; Division of Otolaryngology, Department of Otolaryngology, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; Kuntz N; Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Rao VK; Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Stanley Manne Children's Research Institute, Chicago, IL, USA.

Publisher: BioMed Central Country of Publication: England NLM ID: 101319628 Publication Model: Electronic Cited Medium: Internet ISSN: 1755-8794 (Electronic) Linking ISSN: 17558794 NLM ISO Abbreviation: BMC Med Genomics Subsets: MEDLINE

Krosschell KJ; Department of Physical Therapy and Human Movement Sciences, Northwestern University Feinberg School of Medicine, USA.; Department of Pediatrics, Northwestern University Feinberg School of Medicine, USA.; Brown L; Department of Rehabilitation Services, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Hoffman K; Department of Rehabilitation Services, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Zumpf KB; Biostatistics Collaboration Center, Northwestern University Feinberg School of Medicine, Chicago, IL, USA- sponsor.; Statistical Consulting, OSF Healthcare, Chicago, IL, USA.; Munson H; Chicago College of Osteopathic Medicine, Midwestern University Downers Grove, IL, USA.; Bidwell J; Graduate Program of Genetic Engineering, Northwestern University, Chicago, IL, USA.; Schulte DP; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Schwaede AN; Department of Pediatrics, Northwestern University Feinberg School of Medicine, USA.; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Buehner AN; Ultragenyx Pharmaceutical, Inc, USA.; DiDonato CJ; Department of Pediatrics, Northwestern University Feinberg School of Medicine, USA.; Stanley Manne Children's Research Institute, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Kuntz NL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, USA.; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Rao VK; Department of Pediatrics, Northwestern University Feinberg School of Medicine, USA.; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.; Stanley Manne Children's Research Institute, Ann and Robert H. Lurie Children's Hospital of Chicago, USA.

Publisher: SAGE Publications Country of Publication: United States NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE

Clemens PR; Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.; Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA.; Rao VK; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.; Connolly AM; Division of Neurology, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH, USA.; Harper AD; Children's Hospital of Richmond at Virginia Common wealth University, Richmond, VA, USA.; Mah JK; Department of Pediatrics, University of Calgary, Calgary, Alberta, Canada.; McDonald CM; Department of Physical Medicine and Rehabilitation, Department of Pediatrics, UC Davis Health, University of California, Davis, Sacramento, CA, USA.; Smith EC; Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.; Zaidman CM; Department of Neurology, Washington University at St Louis, St Louis, MO, USA.; Nakagawa T; NS Pharma, Inc., Paramus, NJ, USA.; Hoffman EP; Department of Pharmaceutical Sciences, Binghamton University - State University of New York, Binghamton, NY.

Publisher: SAGE Publications Country of Publication: United States NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE

Diehl E; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois. Electronic address: emily.diehl@seattlechildrens.org.; O'Neill M; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois; Division of Developmental and Behavioral Pediatrics, Ann and Robert Lurie Children's Hospital, Chicago, Illinois.; Gray L; Division of Developmental and Behavioral Pediatrics, Ann and Robert Lurie Children's Hospital, Chicago, Illinois.; Schwaede A; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.; Kuntz N; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

Publisher: Elsevier Science Publishing Country of Publication: United States NLM ID: 8508183 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5150 (Electronic) Linking ISSN: 08878994 NLM ISO Abbreviation: Pediatr Neurol Subsets: MEDLINE

Brandsema JF; Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.; Ginsberg M; Department of Neurology, Akron Children's Hospital, Akron, Ohio.; Hoshino H; Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan.; Mimaki M; Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan.; Nagata S; Department of Pediatrics, Tokyo Women's Medical University School of Medicine, Tokyo, Japan.; Rao VK; Division of Neurology, Lurie Children's Hospital, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.; Ruzhansky K; Department of Neurology, Medical University of South Carolina, Charleston, South Carolina.; Suresh N; Department of Neurology, University of South Florida, Tampa, Florida.; Tiongson E; Division of Neurology, Children's Hospital Los Angeles, Los Angeles, California.; Yamanouchi H; Department of Pediatrics, Comprehensive Epilepsy Center, Saitama Medical University, Saitama, Japan.; Frick G; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts. Electronic address: Glen.Frick@alexion.com.; Hicks E; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts.; Liao S; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts.; Howard JF Jr; Department of Neurology, The University of North Carolina, Chapel Hill, North Carolina.

Publisher: Elsevier Science Publishing Country of Publication: United States NLM ID: 8508183 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5150 (Electronic) Linking ISSN: 08878994 NLM ISO Abbreviation: Pediatr Neurol Subsets: MEDLINE

Veerapandiyan A; Division of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE

Clemens PR; Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.; Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania.; Rao VK; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Connolly AM; Division of Neurology, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio.; Harper AD; Children's Hospital of Richmond at Virginia Commonwealth University, Richmond, Virginia.; Mah JK; Department of Pediatrics, University of Calgary, Calgary, Alberta, Canada.; McDonald CM; Department of Physical Medicine and Rehabilitation, Department of Pediatrics, UC Davis Health, University of California, Davis, Sacramento, California.; Smith EC; Duke University Medical Center, Durham, North Carolina.; Zaidman CM; Department of Neurology, Washington University at St Louis, St Louis, Missouri.; Nakagawa T; NS Pharma, Inc, Paramus, New Jersey.; Hoffman EP; Department of Pharmaceutical Sciences, State University of New York at Binghamton, Binghamton, New York.

Publisher: SAGE Publications Country of Publication: United States NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis 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

Tranel ES; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; McGowan B; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; Drackley A; Division of Genetics, Genomics and Metabolism, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; Epstein LG; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; Rao VK; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; Kuntz NL; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.; Schwaede AN; Division of Neurology, Ann & Robert H. Lurie Children's Hospital, Chicago, IL, United States of America.

Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101624422 Publication Model: eCollection Cited Medium: Print ISSN: 2214-4269 (Print) Linking ISSN: 22144269 NLM ISO Abbreviation: Mol Genet Metab Rep Subsets: PubMed not MEDLINE

De Boer C; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois. Electronic address: chris.deboer09@gmail.com.; Zeineddin S; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Ott K; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Hu A; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Linton S; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; George M; Division of Rheumatology, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania.; Rao VK; Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Abdullah F; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Chin A; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Goldstein SD; Division of Pediatric Surgery, Department of Surgery, Northwestern University Feinberg School of Medicine, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.

Publisher: Elsevier Science Publishing Country of Publication: United States NLM ID: 8508183 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-5150 (Electronic) Linking ISSN: 08878994 NLM ISO Abbreviation: Pediatr Neurol Subsets: MEDLINE

Harada Y; Department of Neurology, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Arya K; Division of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Kuntz NL; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; DiDonato CJ; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Napchan-Pomerantz G; Department of Pediatric Pulmonology, Joe DiMaggio Children's Hospital, Hollywood, Florida, USA.; Agarwal A; Division of Pulmonology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Stefans V; Department of Pediatrics and Physical Medicine and Rehabilitation, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Katsuno M; Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan.; Veerapandiyan A; Division of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.

Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE

Clemens PR; Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.; Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania.; Rao VK; Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Connolly AM; Division of Neurology, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus.; Harper AD; Children's Hospital of Richmond at Virginia Commonwealth University, Richmond.; Mah JK; Department of Pediatrics, University of Calgary, Calgary, Alberta, Canada.; Smith EC; Division of Pediatric Neurology, Duke University Medical Center, Durham, North Carolina.; McDonald CM; Department of Physical Medicine and Rehabilitation, Department of Pediatrics, UC Davis Health, University of California, Davis, Sacramento.; Zaidman CM; Department of Neurology, Washington University at St Louis, St Louis, Missouri.; Morgenroth LP; Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania.; Osaki H; NS Pharma, Paramus, New Jersey.; Satou Y; NS Pharma, Paramus, New Jersey.; Yamashita T; NS Pharma, Paramus, New Jersey.; Hoffman EP; AGADA BioSciences, Dalhousie University, Halifax, Nova Scotia, Canada.; Department of Pharmaceutical Sciences, State University of New York at Binghamton.

Publisher: American Medical Association Country of Publication: United States NLM ID: 101589536 Publication Model: Print Cited Medium: Internet ISSN: 2168-6157 (Electronic) Linking ISSN: 21686149 NLM ISO Abbreviation: JAMA Neurol Subsets: MEDLINE

Veerapandiyan A; Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, Arkansas, USA.; Connolly AM; Department of Pediatrics, Division of Neurology, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio, USA.; Finkel RS; Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.; Arya K; Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, Arkansas, USA.; Mathews KD; Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa city, Iowa, USA.; Smith EC; Department of Pediatrics, Division of Neurology, Duke University Medical Center, Durham, North Carolina, USA.; Castro D; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.; Butterfield RJ; Department of Pediatrics and Neurology, University of Utah School of Medicine, Salt Lake City, Utah, USA.; Parsons JA; Department of Neurology and Pediatrics, University of Colorado School of Medicine, Aurora, Colorado, USA.; Servais L; University of Liège, Neuromuscular Reference Center Disease, Department of Pediatrics, Liege, Belgium.; MDUK Neuromuscular Center, Department of Pediatrics, University of Oxford, United Kingdom.; Kuntz N; Department of Pediatrics, Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Rao VK; Department of Pediatrics, Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Brandsema JF; Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Mercuri E; Pediatric Neurology, Catholic University; Centro Clinico Nemo, Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Ciafaloni E; Department of Neurology, University of Rochester Medical Center, Rochester, New York, USA.

Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE

Veerapandiyan A; Division of Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, Arkansas.; Wagner KR; Center for Genetic Muscle Disorders, Kennedy Krieger Institute and Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, Maryland.; Apkon S; Department of Physical Medicine and Rehabilitation, University of Colorado School of Medicine, Aurora, Colorado.; McDonald CM; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California Davis Health System, Sacramento, California.; Mathews KD; Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa.; Parsons JA; Department of Neurology and Pediatrics, University of Colorado School of Medicine, Aurora, Colorado.; Wong BL; Department of Pediatrics and Neurology, University of Massachusetts Medical School, Worcester, Massachusetts.; Eichinger K; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Shieh PB; Department of Neurology, University of California Los Angeles, Los Angeles, California.; Butterfield RJ; Department of Pediatrics and Neurology, University of Utah School of Medicine, Salt Lake City, Utah.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.; Smith EC; Division of Neurology, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina.; Proud CM; Division of Neurology, Department of Pediatrics, Children's Hospital of The King's Daughters, Norfolk, Virginia.; Connolly AM; Division of Neurology, Department of Pediatrics, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio.; Ciafaloni E; Department of Neurology, University of Rochester Medical Center, Rochester, New York.

Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE

Starr LJ; Department of Pediatrics, Children's Hospital and Medical Center, University of Nebraska Medical Center, Omaha, Nebraska.; Spranger JW; University of Mainz Children's Hospital, Mainz, Germany.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.; Lutz R; Department of Pediatrics, Children's Hospital and Medical Center, University of Nebraska Medical Center, Omaha, Nebraska.; Yetman AT; Department of Pediatrics, Children's Hospital and Medical Center, University of Nebraska Medical Center, Omaha, Nebraska.

Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 101235741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1552-4833 (Electronic) Linking ISSN: 15524825 NLM ISO Abbreviation: Am J Med Genet A Subsets: MEDLINE

Kwon JM; Division of Pediatric Neurology, Department of Neurology, University of Wisconsin-Madison School of Medicine and Public Health, Madison, Wisconsin, USA.; Arya K; Division of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Kuntz N; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.; Phan HC; Rare Disease Research, LLC, Atlanta, Georgia, USA.; Sieburg C; Division of Pediatric Neurology, Department of Neurology, University of Wisconsin-Madison School of Medicine and Public Health, Madison, Wisconsin, USA.; Swoboda KJ; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Veerapandiyan A; Division of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.; Assman B; Genentech, South San Francisco, California, USA.; Bader-Weder S; F. Hoffmann-La Roche Ltd, Basel, Switzerland.; Dickendesher TL; Genentech, South San Francisco, California, USA.; Hansen J; Genentech, South San Francisco, California, USA.; Lin H; Genentech, South San Francisco, California, USA.; Yan Y; Genentech, South San Francisco, California, USA.; Rao VK; Division of Neurology, Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

Publisher: Wiley Periodicals, Inc on behalf of American Neurological Association Country of Publication: United States NLM ID: 101623278 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2328-9503 (Electronic) Linking ISSN: 23289503 NLM ISO Abbreviation: Ann Clin Transl Neurol Subsets: MEDLINE