Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy.

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作者： Craig M,McDonald ^[1] ; Jacob S,Elkins ^[2] ; Sai,Dharmarajan ^[2] ; Katherine,Gooch ^[2] ; Teofil,Ciobanu ^[3] ; Claire J,Lansdall ^[3] ; Alexander P,Murphy ^[4] ; Fiona,McDougall ^[5] ; Eugenio M,Mercuri ^[6] ; Ivana,Audhya ^[7] ; EMBARK Study Group

作者单位： University of California, 4860 Y St #1700, Sacramento, CA, 95819, USA. cmmcdonald@ucdavis.edu. ^[1] Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA, 02142, USA. ^[2] F. Hoffmann-La Roche Ltd., Basel, Switzerland. ^[3] F. Hoffmann-La Roche Ltd, Welwyn Garden City, UK. ^[4] Genentech, South San Francisco, CA, 94080, USA. ^[5] Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy. ^[6] Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA, 02142, USA. iaudhya@sarepta.com. ^[7]

关键词 Caregiver Delandistrogene moxeparvovec Duchenne muscular dystrophy Gene therapy Global Impressions scale

DOI 10.1007/s40120-024-00685-8

PMID 39589719

发布时间 2024-11-26

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Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy.

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Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy.

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Neurology and therapy

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