Neuropediatrics 2025; 56(S 01): S1-S24
DOI: 10.1055/s-0045-1812122
DOI: 10.1055/s-0045-1812122
Neuromuscular Disorders
Long-Term Functional Outcomes, Safety, and Micro-Dystrophin Expression Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results
Authors
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I. Seelmann
1 Uniklinik Essen, Essen, Germany -
J. R. Mendell
2 Sarepta Therapeutics, Inc., Cambridge, United States -
F. Muntoni
3 Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health and Institute of Neurology, University College London, & Great Ormond Street Hospital Trust, London, United Kingdom -
C. M. McDonald
4 UC Davis Health, Sacramento, United States -
E. Mercuri
5 Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy -
E. Ciafaloni
6 University of Rochester Medical Center, Rochester, United States -
H. Komaki
7 Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan -
C. Leon-Astudillo
8 Department of Pediatrics, University of Florida, Gainesville, Florida, United States -
A. Nascimento
9 Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Déu, CIBERER, ISC III, Barcelona, Spain -
C. Proud
10 Children's Hospital of the King's Daughters, Norfolk, United States -
U. Schara-Schmidt
11 Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany -
A. Veerapandiyan
12 Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, United States -
C. M. Zaidman
13 Department of Neurology, Washington University in St Louis, St Louis, United States -
M. Furgerson
2 Sarepta Therapeutics, Inc., Cambridge, United States -
K. Ding
2 Sarepta Therapeutics, Inc., Cambridge, United States -
P. Singh
2 Sarepta Therapeutics, Inc., Cambridge, United States -
R. Potter
2 Sarepta Therapeutics, Inc., Cambridge, United States -
D. R. Asher
2 Sarepta Therapeutics, Inc., Cambridge, United States -
A. P. Murphy
14 Roche Products Ltd, Welwyn Garden City, United Kingdom -
C. Reid
14 Roche Products Ltd, Welwyn Garden City, United Kingdom -
G. Hooper
14 Roche Products Ltd, Welwyn Garden City, United Kingdom -
C. O. Torre
14 Roche Products Ltd, Welwyn Garden City, United Kingdom -
M. Manfrini
15 F. Hoffmann-La Roche Ltd, Basel, Switzerland -
J. S. Elkins
2 Sarepta Therapeutics, Inc., Cambridge, United States -
L. R. Rodino-Klapac
2 Sarepta Therapeutics, Inc., Cambridge, United States -
on behalf of the EMBARK Study Group
