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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.
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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  Data: Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.
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  Data: <searchLink fieldCode="AU" term="%22Mendell+JR%22">Mendell JR</searchLink>; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA. JMendell@sarepta.com.; Sarepta Therapeutics, Inc, Cambridge, MA, USA. JMendell@sarepta.com.<br /><searchLink fieldCode="AU" term="%22Muntoni+F%22">Muntoni F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22McDonald+CM%22">McDonald CM</searchLink>; UC Davis Health, Sacramento, CA, USA.<br /><searchLink fieldCode="AU" term="%22Mercuri+EM%22">Mercuri EM</searchLink>; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Ciafaloni+E%22">Ciafaloni E</searchLink>; University of Rochester Medical Center, Rochester, NY, USA.<br /><searchLink fieldCode="AU" term="%22Komaki+H%22">Komaki H</searchLink>; Translational Medical Center, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Leon-Astudillo+C%22">Leon-Astudillo C</searchLink>; Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Nascimento+A%22">Nascimento A</searchLink>; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Déu, CIBERER - ISC III, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Proud+C%22">Proud C</searchLink>; Children's Hospital of the King's Daughters, Norfolk, VA, USA.<br /><searchLink fieldCode="AU" term="%22Schara-Schmidt+U%22">Schara-Schmidt U</searchLink>; Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany.<br /><searchLink fieldCode="AU" term="%22Veerapandiyan+A%22">Veerapandiyan A</searchLink>; Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA.<br /><searchLink fieldCode="AU" term="%22Zaidman+CM%22">Zaidman CM</searchLink>; Department of Neurology, Washington University in St Louis, St Louis, MO, USA.<br /><searchLink fieldCode="AU" term="%22Furgerson+M%22">Furgerson M</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ding+K%22">Ding K</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Singh+P%22">Singh P</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Potter+R%22">Potter R</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Asher+DR%22">Asher DR</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Murphy+AP%22">Murphy AP</searchLink>; Roche Products Ltd, Welwyn Garden City, UK.<br /><searchLink fieldCode="AU" term="%22Reid+C%22">Reid C</searchLink>; Roche Products Ltd, Welwyn Garden City, UK.<br /><searchLink fieldCode="AU" term="%22Hooper+G%22">Hooper G</searchLink>; Roche Products Ltd, Welwyn Garden City, UK.<br /><searchLink fieldCode="AU" term="%22Torre+CO%22">Torre CO</searchLink>; Roche Products Ltd, Welwyn Garden City, UK.<br /><searchLink fieldCode="AU" term="%22Manfrini+M%22">Manfrini M</searchLink>; F. Hoffmann-La Roche Ltd, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Rodino-Klapac+LR%22">Rodino-Klapac LR</searchLink>; Sarepta Therapeutics, Inc, Cambridge, MA, USA.
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  Data: <searchLink fieldCode="JN" term="%22101637818%22">Neurology and therapy</searchLink> [Neurol Ther] 2026 Apr; Vol. 15 (2), pp. 545-559. <i>Date of Electronic Publication: </i>2026 Jan 10.
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