[O9] 末梢神経・筋③

[O9-05] EMBARK Part 1 3年およびPart2 2年データ：DMDにおけるdelandistrogene moxeparvovecの有効性および安全性

石垣 景子¹, Mendell Jerry R.², Muntoni Francesco³, Mercuri Eugenio M.⁴, Schara-Schmidt Ulrike⁵, Furgerson Matthew², Murphy Alexander P.⁶, Reid Carol⁶, Rodino-Klapac Louise R.² (1.東京女子医科大学医学部小児科, 2.Sarepta Therapeutics, Inc., Cambridge, MA, USA, 3.The Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health University College London, London, UK, 4.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 5.Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany, 6.Roche Products Ltd, Welwyn Garden City, UK)

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