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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
Ann Neurol. 2023.
PMID: 37539981
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR.
Mendell JR, et al. Among authors: darton e.
Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14.
Muscle Nerve. 2024.
PMID: 37577753
Clinical Trial.
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR.
Mendell JR, et al. Among authors: darton e.
Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9.
Nat Med. 2025.
PMID: 39385046
Free PMC article.
Clinical Trial.
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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy.
Mendell JR, Proud C, Zaidman CM, Mason S, Darton E, Wang S, Wandel C, Murphy AP, Mercuri E, Muntoni F, McDonald CM.
Mendell JR, et al. Among authors: darton e.
Pediatr Neurol. 2024 Apr;153:11-18. doi: 10.1016/j.pediatrneurol.2024.01.003. Epub 2024 Jan 5.
Pediatr Neurol. 2024.
PMID: 38306745
Free article.
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