Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.
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| Title: | Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. |
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| Authors: | Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA. JMendell@sarepta.com.; Sarepta Therapeutics, Inc, Cambridge, MA, USA. JMendell@sarepta.com., Muntoni F; 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, UK., McDonald CM; UC Davis Health, Sacramento, CA, USA., Mercuri EM; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Ciafaloni E; University of Rochester Medical Center, Rochester, NY, USA., Komaki H; Translational Medical Center, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan., Leon-Astudillo C; Department of Pediatrics, University of Florida, Gainesville, FL, USA., Nascimento A; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Déu, CIBERER - ISC III, Barcelona, Spain., Proud C; Children's Hospital of the King's Daughters, Norfolk, VA, USA., Schara-Schmidt U; Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany., Veerapandiyan A; Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA., Zaidman CM; Department of Neurology, Washington University in St Louis, St Louis, MO, USA., Furgerson M; Sarepta Therapeutics, Inc, Cambridge, MA, USA., Ding K; Sarepta Therapeutics, Inc, Cambridge, MA, USA., Singh P; Sarepta Therapeutics, Inc, Cambridge, MA, USA., Potter R; Sarepta Therapeutics, Inc, Cambridge, MA, USA., Asher DR; Sarepta Therapeutics, Inc, Cambridge, MA, USA., Murphy AP; Roche Products Ltd, Welwyn Garden City, UK., Reid C; Roche Products Ltd, Welwyn Garden City, UK., Hooper G; Roche Products Ltd, Welwyn Garden City, UK., Torre CO; Roche Products Ltd, Welwyn Garden City, UK., Manfrini M; F. Hoffmann-La Roche Ltd, Basel, Switzerland., Rodino-Klapac LR; Sarepta Therapeutics, Inc, Cambridge, MA, USA. |
| Source: | Neurology and therapy [Neurol Ther] 2026 Apr; Vol. 15 (2), pp. 545-559. Date of Electronic Publication: 2026 Jan 10. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Springer Country of Publication: New Zealand NLM ID: 101637818 Publication Model: Print-Electronic Cited Medium: Print ISSN: 2193-8253 (Print) Linking ISSN: 21936536 NLM ISO Abbreviation: Neurol Ther Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2193-8253 |
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| DOI: | 10.1007/s40120-025-00879-8 |
