Presentation Information
[O15-03]EMBARK Part 2: Safety and efficacy of delandistrogene moxeparvovec in duchenne muscular dystrophy
○Hirofumi Komaki1, Jerry R. Mendell2,3, Francesco Muntoni4, Eugenio Mercuri5, Ulrike Schara-Schmidt6, Alex Murphy7, Carol Reid7, Jacob S. Elkins8, Louise R. Rodino-Klapac8 (1.Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan, 2.Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA, 3.The Ohio State University, Columbus, OH, USA, 4.The Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health University College London, London, UK, 5.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 6.Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany, 7.Roche Products Ltd, Welwyn Garden City, UK, 8.Sarepta Therapeutics, Inc., Cambridge, MA, USA)