부산대학교 도서관

Alfano, Lindsay N; Eagle, Michelle; James, Meredith K; Lowes, Linda; Mayhew, Anna; Mazzone, Elena S; Nelson, Leslie; Rose, Kristy J; Abdel-Hamid, Hoda Z; Apkon, Susan D; Barohn, Richard J; Bertini, Enrico; Bloetzer, Clemens; de Vaud, Lausanne Canton; Butterfield, Russell J; Chabrol, Brigitte; Chae, Jong-Hee; Jongno-gu, Daehak-ro; Comi, Giacomi Pietro; Darras, Basil T; Dastgir, Jahannaz; Desguerre, Isabelle; Escobar, Raul G; Finanger, Erika; Guglieri, Michela; Hughes, Imelda; Iannaccone, Susan T; Jones, Kristi J; Karachunski, Peter; Kudr, Martin; Lotze, Timothy; Mah, Jean K; Mathews, Katherine; Nevo, Yoram; Parsons, Julie; Péréon, Yann; de Queiroz Campos Araujo, Alexandra Prufer; Renfroe, J Ben; de Resende, Maria Bernadete Dutra; Ryan, Monique; Selby, Kathryn; Tennekoon, Gihan; Vita, Giuseppe; McDonald, Craig M ; Campbell, Craig ; Torricelli, Ricardo Erazo ; Finkel, Richard S ; Flanigan, Kevin M ; Goemans, Nathalie ; Heydemann, Peter ; Kaminska, Anna ; Kirschner, Janbernd ; Muntoni, Francesco ; Osorio, Andrés Nascimento ; Schara, Ulrike ; Sejersen, Thomas ; Shieh, Perry B ; Sweeney, H Lee ; Topaloglu, Haluk ; Tulinius, Már ; Vilchez, Juan J ; Voit, Thomas ; Wong, Brenda ; Elfring, Gary ; Kroger, Hans ; Luo, Xiaohui ; McIntosh, Joseph ; Ong, Tuyen ; Riebling, Peter ; Souza, Marcio ; Spiegel, Robert J ; Peltz, Stuart W ; Mercuri, Eugenio

In The Lancet 23-29 September 2017 390(10101):1489-1498

Mendell, Jerry R ; Sahenk, Zarife ; Malik, Vinod ; Gomez, Ana M ; Flanigan, Kevin M ; Lowes, Linda P ; Alfano, Lindsay N ; Berry, Katherine ; Meadows, Eric ; Lewis, Sarah ; Braun, Lyndsey ; Shontz, Kim ; Rouhana, Maria ; Clark, Kelly Reed ; Rosales, Xiomara Q ; Al-Zaidy, Samiah ; Govoni, Alessandra ; Rodino-Klapac, Louise R ; Hogan, Mark J ; Kaspar, Brian K

In Molecular Therapy January 2015 23(1):192-201

Wallace, Lindsay M ; Liu, Jian ; Domire, Jacqueline S ; Garwick-Coppens, Sara E ; Guckes, Susan M ; Mendell, Jerry R ; Flanigan, Kevin M ; Harper, Scott Q.

In Molecular Therapy July 2012 20(7):1417-1423

Drakou E; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio.; Needum C; Gene Therapy Center of Excellence, Nationwide Children's Hospital, Columbus, Ohio.; Konst K; Gene Therapy Center of Excellence, Nationwide Children's Hospital, Columbus, Ohio.; Chagat S; Gene Therapy Center of Excellence, Nationwide Children's Hospital, Columbus, Ohio.; Division of Neurology, Nationwide Children's Hospital, Columbus, Ohio.; Abraham RS; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pathology, The Ohio State University College of Medicine, Columbus, Ohio.; Weymann A; Division of Gastroenterology, Hepatology and Nutrition, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.; Flanigan KM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio.; Gene Therapy Center of Excellence, Nationwide Children's Hospital, Columbus, Ohio.; Division of Neurology, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.; Department of Neurology, The Ohio State University College of Medicine, Columbus, Ohio.

Publisher: American Academy of Pediatrics Country of Publication: United States NLM ID: 0376422 Publication Model: Print Cited Medium: Internet ISSN: 1098-4275 (Electronic) Linking ISSN: 00314005 NLM ISO Abbreviation: Pediatrics Subsets: MEDLINE

Flanigan, Kevin M ; Coffeen, Christin M ; Sexton, Lee ; Stauffer, Dora ; Brunner, Shari ; Leppert, Mark F

In Neuromuscular Disorders 2001 11(6):525-529

Flanigan, Kevin M

In The Lancet Neurology July 2016 15(8):785-787

Peay H; RTI Health Solutions, Research Triangle Park, NC, USA. Electronic address: holly.peay@faegredrinker.com.; Fischer R; Parent Project Muscular Dystrophy, 1012 14th NW, Suite 500, Washington, D.C., 20005, USA.; McNiff M; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Electronic address: megan.mcniff@ncl.ac.uk.; Heslop E; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.; Pierce A; RTI Health Solutions, Research Triangle Park, NC, USA.; Denger B; Parent Project Muscular Dystrophy, 1012 14th NW, Suite 500, Washington, D.C., 20005, USA.; Camino E; Parent Project Muscular Dystrophy, 1012 14th NW, Suite 500, Washington, D.C., 20005, USA.; Johnson A; Duchenne UK, London, UK.; Cope H; RTI Health Solutions, Research Triangle Park, NC, USA.; Hill C; RTI Health Solutions, Research Triangle Park, NC, USA.; Beaverson KL; Pfizer, Inc., New York, NY, 10018, USA.; Ganot A; Solid Biosciences Inc., Charlestown, MA, USA.; Phillips D; Regenxbio Inc., Rockville, MD, USA.; Woollacott IOC; Pfizer Ltd., Walton Oaks, Dorking Road, Tadworth, Surrey, UK.; Bateman-House A; Division of Medical Ethics, Department of Population Health, NYU Grossman School of Public Health, New York, NY, USA.; Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, USA; Department of Pediatrics and Neurology, The Ohio State University, USA.; Goemans N; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium.; Servais L; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK; NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium.; Guglieri M; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.; Mansfield C; RTI Health Solutions, Research Triangle Park, NC, USA.

Publisher: Elsevier Country of Publication: Netherlands NLM ID: 101247089 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-0849 (Electronic) Linking ISSN: 17697212 NLM ISO Abbreviation: Eur J Med Genet Subsets: MEDLINE

Taylor A; Department of Neurology, University of Pennsylvania, USA.; Yacob D; College of Medicine, The Ohio State University, USA; Center for Colorectal and Pelvic Reconstruction, Nationwide Children's Hospital, USA; Department of Pediatrics, Duke University Medical Center, USA.; Fung B; Department of Pediatrics, The Ohio State University, USA; Department of Pathology & Laboratory Medicine, University of Calgary, Canada.; Mangray S; College of Medicine, The Ohio State University, USA; Pathology and Laboratory Medicine, Nationwide Children's Hospital, USA; Department of Pathology, The Ohio State University, USA.; Boué DR; College of Medicine, The Ohio State University, USA; Pathology and Laboratory Medicine, Nationwide Children's Hospital, USA; Department of Pathology, The Ohio State University, USA.; Flanigan KM; College of Medicine, The Ohio State University, USA; Center for Gene Therapy, Nationwide Children's Hospital, USA; Department of Pediatrics, The Ohio State University, USA.; Koch RL; Department of Pediatrics, Duke University Medical Center, USA.; Kishnani PS; Department of Pediatrics, Duke University Medical Center, USA.; Bali D; Department of Pediatrics, Duke University Medical Center, USA.; Weymann A; College of Medicine, The Ohio State University, USA; Gastroenterology & Hepatology & Nutrition, Nationwide Children's Hospital, USA; Department of Pediatrics, The Ohio State University, USA.; Mori M; Department of Pediatrics, The Ohio State University, USA; Department of Human Genetics, Emory University School of Medicine, USA. Electronic address: mari.mori@emory.edu.

Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE

Lowes LP; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Reash NF; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Iammarino MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Connolly AM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Pietruszewski L; Center for Cerebral Palsy, University of California Los Angeles, Los Angeles, California, USA.; Smith MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Peng J; Center for Biostatistics, Department of Biomedical Informatics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Steiner CL; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Tsao CY; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Waldrop MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Flanigan KM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Chagat S; Department of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.; Meyer AP; Division of Genetic and Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Neurology, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Sarepta Therapeutics, Cambridge, MA, USA.; Alfano LN; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.

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

De Winter, Jonathan; Van de Vondel, Liedewei; Ermanoska, Biljana; Monticelli, Alice; Isapof, Arnaud; Cohen, Enzo; Stojkovic, Tanya; Hackman, Peter; Johari, Mridul; Palmio, Johanna; Waldrop, Megan A; Meyer, Alayne P; Nicolau, Stefan; Flanigan, Kevin M; Töpf, Ana; Diaz-Manera, Jordi; Straub, Volker; Longman, Cheryl; McWilliam, Catherine A.; Orbach, Rotem; Verma, Sumit; Laine, Regina; Donkervoort, Sandra; Bonnemann, Carsten G.; Rebelo, Adriana; Züchner, Stephan; Grider, Tiffany; Shy, Michael E.; Maystadt, Isabelle; Demurger, Florence; Cairns, Anita; Beecroft, Sarah; Folland, Chiara; De Ridder, Willem; Ravenscroft, Gina; Bonne, Gisèle; Udd, Bjarne; Baets, Jonathan

medRxiv : the preprint server for health sciences

Wolff JM; Parent Project Muscular Dystrophy, USA.; Capocci N; Muscular Dystrophy Association, USA.; Atas E; Muscular Dystrophy Association, USA.; Bharucha-Goebel DX; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA, Division of Neurology, Children's National Hospital, WA, DC, USA.; Brandsema JF; Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, PA, USA.; Butterfield RJ; Department of Pediatric Neurology, University of Utah, Salt Lake City, UT, USA.; Chadwick CB; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.; Corti M; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.; Crawford TO; Department of Pediatrics, Johns Hopkins University, Baltimore, MD, USA.; Cripe L; Department of Pediatrics and Neurology, Nationwide Children's Hopsital, Columbus, OH, USA.; Day JW; Department of Neurology, Stanford University, Stanford, CA, USA.; Duong T; Department of Neurology, Stanford University, Stanford, CA, USA.; ElMallah MK; Department of Pediatrics, School of Medicine, Duke University, Durham, NC, USA.; Flanigan KM; Department of Pediatrics and Neurology, Nationwide Children's Hopsital, Columbus, OH, USA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; George LA; Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia PA, USA.; Goedeker NL; Department of Neurology, Washington University in St. Louis School of Medicine, St. Louis, MO, USA.; Goude E; Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Sacramento, CA, USA.; Hesterlee S; Muscular Dystrophy Association, USA.; Lin B; Muscular Dystrophy Association, USA.; Katz NK; Department of Pediatrics, School of Medicine, Duke University, Durham, NC, USA.; Matesanz SE; Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, PA, USA.; McDonald C; Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Sacramento, CA, USA.; McNally EM; Center for Genetic Medicine, Bluhm Cardiovascular Institute, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; Mercado-Rodriguez C; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.; Nandi D; The Heart Center, Nationwide Children's Hospital, Columbus, OH, USA.; Parsons JA; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA.; Proud C; Children's Hospital of the King's Daughters, Norfolk, VA, USA.; Ramos-Platt L; Department of Pediatrics, Keck School of Medicine, University of Southern California and Children's Hospital of Los Angeles, Los Angeles, CA, USA.; Lek A; Muscular Dystrophy Association, USA.; Salabarria SM; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.; Camino E; Parent Project Muscular Dystrophy, USA.; Schrader R; Parent Project Muscular Dystrophy, USA.; Shea E; Department of Pediatrics, School of Medicine, Duke University, Durham, NC, USA.; Shell R; Department of Pediatrics and Neurology, Nationwide Children's Hopsital, Columbus, OH, USA.; Shieh PB; Department of Pediatrics, University of California, Los Angeles, CA USA.; Soslow JH; Division of Pediatric Cardiology, Vanderbilt University Medical Center, Nashville, TN, USA.; Taylor JB; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.; Veerapandiyan A; Department of Pediatrics, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA.; Villa C; The Heart Institute, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Yang ML; Department of Pediatrics, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA.; Zaidman CM; Department of Neurology, Washington University in St. Louis School of Medicine, St. Louis, MO, USA.; Leon-Astudillo C; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.; Byrne BJ; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA. Electronic address: Barry.byrne@ufl.edu.

Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE

Meyer AP; Division of Genetic and Genomic Medicine, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA. Electronic address: Alayne.Meyer@nationwidechildrens.org.; Yousfi S; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA.; Nicolau S; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA.; Rashnonejad A; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA.; Zhu J; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA.; Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA; Department of Neurology, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA.; Frair E; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA.; Lin H; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA.; Ramadesikan S; The Steve and Cindy Rasmussen Institute for Genomic Medicine, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.; Koboldt D; Department of Pediatrics, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA; The Steve and Cindy Rasmussen Institute for Genomic Medicine, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.; Flanigan KM; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's 700 Children's Drive, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA; Department of Neurology, The Ohio State University College of Medicine, 1645 Neil Avenue, Columbus, OH 43210, USA.

Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE

Tsuda, Takeshi; Fitzgerald, Kristi; Scavena, Mena; Gidding, Samuel; Cox, Mary O; Marks, Harold; Flanigan, Kevin M; Moore, Steven A

Journal of Human Genetics. Mar2015, Vol. 60 Issue 3, p151-155. 5p.

Nicolau S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Malhotra J; Sarepta Therapeutics Inc., Cambridge, MA, USA.; Kaler M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Magistrado-Coxen P; Sarepta Therapeutics Inc., Cambridge, MA, USA.; Iammarino MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Reash NF; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Frair EC; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Wijeratne S; Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, USA.; Kelly BJ; Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, USA.; White P; Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.; Lowes LP; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Waldrop MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.; >Department of Neurology>, The Ohio State University, Columbus, OH, USA.; Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.; >Department of Neurology>, The Ohio State University, Columbus, OH, 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

Byrne BJ; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA. Electronic address: barry.byrne@ufl.edu.; Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.; Matesanz SE; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Philadelphia, PA, USA.; Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.; Waldrop MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.; D'Ambrosio ES; Department of Neurology, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.; Johnson NE; Department of Neurology and Center for Inherited Muscle Research, Virginia Commonwealth University, Richmond, VA, USA.; Smith BK; Department of Physical Therapy, College of Public Health and Health Professions, University of Florida Gainesville, FL, USA.; Bönnemann C; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA.; Carrig S; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Rossano JW; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA; Division of Cardiology, Children's Hospital of Philadelphia, Philadelphia PA, USA.; Greenberg B; Cardiology Department, University of California, California, San Diego, La Jolla, CA, USA.; Lalaguna L; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain.; Lara-Pezzi E; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain; Centro de Investigación Biomédica en Red Cardiovascular (CIBERCV), Madrid, Spain.; Subramony S; Department of Neurology, University of Florida College of Medicinem, Gainesville, FL, USA.; Corti M; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.; Mercado-Rodriguez C; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.; Leon-Astudillo C; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.; Ahrens-Nicklas R; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA.; Bharucha-Goebel D; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA; Division of Neurology, Children's National Hospital, Washington, DC, USA.; Gao G; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.; Gessler DJ; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.; Hwu WL; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan; Center for Precision Medicine, China Medical University Hospital, Taichung, Taiwan.; Chien YH; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan.; Lee NC; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan.; Boye SL; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.; Boye SE; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.; George LA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. Electronic address: georgel@chop.edu.

Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

De Winter J; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium; Neuromuscular Reference Centre, Department of Neurology, Antwerp University Hospital, Antwerp, Belgium.; Van de Vondel L; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium.; Ermanoska B; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium.; Monticelli A; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium.; Isapof A; Centre de Référence des Maladies Neuromusculaires Nord-Est-Ile de France, Hôpital Armand Trousseau, APHP, Paris, France.; Cohen E; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France.; Stojkovic T; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France; Centre de Référence des Maladies Neuromusculaires Nord-Est-Ile de France, Hôpital Pitié-Salpêtrière, Institut de Myologie, APHP, Paris, France.; Hackman P; Folkhälsan Research Center, Helsinki, Finland and Medicum, University of Helsinki, Helsinki, Finland.; Johari M; Folkhälsan Research Center, Helsinki, Finland and Medicum, University of Helsinki, Helsinki, Finland; Centre for Medical Research, University of Western Australia, Harry Perkins Institute of Medical Research, Perth, Western Australia, Australia.; Palmio J; Tampere Neuromuscular Center, Tampere University and Tampere University Hospital Tampere, Finland.; Waldrop MA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Departments of Pediatrics and Neurology, Wexner Medical Center, Ohio State University, Columbus OH.; Meyer AP; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Division of Genetic and Genomic Medicine, Nationwide Children's Hospital, Columbus, OH.; Nicolau S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Department of Pediatrics, The Ohio State University, Columbus, OH.; Flanigan KM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH; Departments of Pediatrics and Neurology, Wexner Medical Center, Ohio State University, Columbus OH.; Töpf A; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle, United Kingdom.; Diaz-Manera J; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle, United Kingdom.; Straub V; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle, United Kingdom.; Longman C; West Scotland Regional Genetics Service, Queen Elizabeth University Hospital, Glasgow, Scotland.; McWilliam CA; West Scotland Regional Genetics Service, Queen Elizabeth University Hospital, Glasgow, Scotland.; Orbach R; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurologic Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Verma S; Department of Pediatrics and Neurology, Emory University School of Medicine, Atlanta, GA.; Laine R; Department of Neurology, Boston Children's Hospital, Boston, MA.; Donkervoort S; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurologic Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Bonnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurologic Disorders and Stroke, National Institutes of Health, Bethesda, MD.; Rebelo A; Dr. John T. Macdonald Foundation Department of Human Genetics and John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL.; Züchner S; Dr. John T. Macdonald Foundation Department of Human Genetics and John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL.; Grider T; Neurology, The University of Iowa Roy J and Lucille A Carver College of Medicine, Iowa City, IA.; Shy ME; Neurology, The University of Iowa Roy J and Lucille A Carver College of Medicine, Iowa City, IA.; Maystadt I; Centre de Génétique Humaine, Institut de Pathologie et de Génétique, Gosselies, Belgium; URPHYM, Department of Medicine, UNamur, Namur, Belgium.; Demurger F; Service de Génétique, CHBA, Vannes, France.; Cairns A; Neurosciences Department, Queensland Children's Hospital, Brisbane, QLD, Australia.; Beecroft S; Pawsey Supercomputing Research Centre, Kensington, WA, Australia.; Folland C; Centre for Medical Research, University of Western Australia, Harry Perkins Institute of Medical Research, Perth, Western Australia, Australia.; De Ridder W; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium; Neuromuscular Reference Centre, Department of Neurology, Antwerp University Hospital, Antwerp, Belgium.; Ravenscroft G; Centre for Medical Research, University of Western Australia, Harry Perkins Institute of Medical Research, Perth, Western Australia, Australia.; Bonne G; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France.; Udd B; Folkhälsan Research Center, Helsinki, Finland and Medicum, University of Helsinki, Helsinki, Finland; Tampere Neuromuscular Center, Tampere University and Tampere University Hospital Tampere, Finland.; Baets J; Translational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium; Laboratory of Neuromuscular Pathology, Institute Born-Bunge, University of Antwerp, Antwerp, Belgium; Neuromuscular Reference Centre, Department of Neurology, Antwerp University Hospital, Antwerp, Belgium. Electronic address: jonathan.baets@uantwerpen.be.

Publisher: Elsevier Country of Publication: United States NLM ID: 9815831 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1530-0366 (Electronic) Linking ISSN: 10983600 NLM ISO Abbreviation: Genet Med Subsets: MEDLINE

Meyer AP; Division of Genetic and Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; Ma J; Department of Biomedical Informatics, The Ohio State University, Columbus, Ohio, USA.; Brock G; Department of Biomedical Informatics, The Ohio State University, Columbus, Ohio, USA.; Hashimoto S; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Cottrell CE; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pathology, The Ohio State University, Columbus, Ohio, USA.; Mathew M; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pathology, The Ohio State University, Columbus, Ohio, USA.; Hunter JM; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pathology, The Ohio State University, Columbus, Ohio, USA.; Leung ML; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pathology, The Ohio State University, Columbus, Ohio, USA.; Corsmeier D; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Jayaraman V; The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.; Waldrop MA; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; Department of Neurology, Nationwide Children's Hospital & The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.; Flanigan KM; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.; Department of Neurology, Nationwide Children's Hospital & The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, 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

Bayazit MB; Henderson D; Nguyen KT; Reátegui E; Tawil R; Flanigan KM; Harper SQ; Saad NY

Country of Publication: United States NLM ID: 101767986 Publication Model: Electronic Cited Medium: Internet NLM ISO Abbreviation: medRxiv Subsets: PubMed not MEDLINE

Guglieri M; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England.; Bushby K; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England.; McDermott MP; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, New York.; Hart KA; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Tawil R; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Martens WB; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Herr BE; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; McColl E; Newcastle University, Newcastle upon Tyne, England.; Speed C; Newcastle University, Newcastle upon Tyne, England.; NIHR Clinical Research Network North East and North Cumbria, Newcastle upon Tyne, England.; Wilkinson J; Newcastle University, Newcastle upon Tyne, England.; Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University Hospital Freiburg, Freiburg, Germany.; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.; King WM; Ohio State University, Columbus.; Eagle M; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England.; Brown MW; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Willis T; Robert Jones and Agnes Hunt Orthopaedic Hospital, NHS Foundation Trust, Oswestry, England.; Griggs RC; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Straub V; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England.; van Ruiten H; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England.; Childs AM; Leeds General Infirmary, Leeds, England.; Ciafaloni E; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Shieh PB; David Geffen School of Medicine, University of California, Los Angeles.; Spinty S; Alderhey Children's Hospital NHS Foundation Trust, Liverpool, England.; Maggi L; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Baranello G; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Dubowitz Neuromuscular Centre, UCL NIHR GOSH Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, London, England.; Butterfield RJ; Department of Pediatric Neurology, University of Utah, Salt Lake City.; Horrocks IA; Glasgow Paediatric Neuromuscular Research Centre, Greater Glasgow and Clyde NHS Yorkhill Hospital, Glasgow, Scotland.; Roper H; University Hospitals Birmingham NHS Foundation Trust, Birmingham, England.; Alhaswani Z; University Hospitals Birmingham NHS Foundation Trust, Birmingham, England.; Flanigan KM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio.; Kuntz NL; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.; Manzur A; Great Ormond Street Hospital, London, England.; Darras BT; Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.; Kang PB; Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis.; Morrison L; Health Sciences Center, University of New Mexico, Albuquerque.; Krzesniak-Swinarska M; Health Sciences Center, University of New Mexico, Albuquerque.; Mah JK; Cumming School of Medicine, University of Calgary and Alberta Children's Hospital Research Institute, Calgary, Canada.; Mongini TE; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Turin, Italy.; Ricci F; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Turin, Italy.; von der Hagen M; Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.; Finkel RS; Nemours Children's Hospital, Orlando, Florida.; Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital, Memphis, Tennessee.; O'Reardon K; Nemours Children's Hospital, Orlando, Florida.; Wicklund M; Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania.; Department of Neurology, School of Medicine, University of Colorado, Aurora.; Kumar A; Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania.; McDonald CM; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento.; Han JJ; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento.; Joyce N; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento.; Henricson EK; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento.; Schara-Schmidt U; Clinic for Pediatrics I, Pediatric Neurology, University Hospital Essen, Essen, Germany.; Gangfuss A; Clinic for Pediatrics I, Pediatric Neurology, University Hospital Essen, Essen, Germany.; Wilichowski E; Department of Pediatrics and Adolescent Medicine, Division of Pediatric Neurology, University Medical Center Göttingen, Göttingen, Germany.; Barohn RJ; University of Missouri, Columbia.; Statland JM; University of Kansas Medical Center, Kansas City.; Campbell C; Departments of Pediatrics, Clinical Neurological Sciences, and Epidemiology, University of Western Ontario, London, Canada.; Vita G; ERN Neuromuscular Unit, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Vita GL; Unit of Neurology, IRCCS Centro Neurolesi Bonino-Pulejo, Messina, Italy.; Howard JF Jr; School of Medicine, University of North Carolina, Chapel Hill.; Hughes I; Royal Manchester Children's Hospital, Manchester, England.; McMillan HJ; Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada.; Pegoraro E; ERN Neuromuscular Unit, Department of Neuroscience, University of Padova, Padua, Italy.; Bello L; ERN Neuromuscular Unit, Department of Neuroscience, University of Padova, Padua, Italy.; Burnette WB; Vanderbilt University Medical Center, Nashville, Tennessee.; Thangarajh M; Department of Neurology, Virginia Commonwealth University, Richmond.; Chang T; Children's National Hospital, George Washington University, Washington, DC.

Publisher: American Medical Association Country of Publication: United States NLM ID: 7501160 Publication Model: Print Cited Medium: Internet ISSN: 1538-3598 (Electronic) Linking ISSN: 00987484 NLM ISO Abbreviation: JAMA Subsets: MEDLINE

Vetter TA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Nicolau S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Bradley AJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Frair EC; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Departments of Pediatrics and Neurology, Ohio State University Wexner Medical Center, Columbus, OH, USA.

Publisher: Blackwell Scientific Publications Country of Publication: England NLM ID: 7609829 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2990 (Electronic) Linking ISSN: 03051846 NLM ISO Abbreviation: Neuropathol Appl Neurobiol Subsets: MEDLINE