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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).
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Zaidman CM, et al. Among authors: murphy ap. Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7. Ann Neurol. 2023. PMID: 37539981
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: murphy ap. 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.
Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy.
McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I; EMBARK Study Group. McDonald CM, et al. Among authors: murphy ap. Neurol Ther. 2024 Nov 26. doi: 10.1007/s40120-024-00685-8. Online ahead of print. Neurol Ther. 2024. PMID: 39589719
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy.
Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Potter RA, et al. Among authors: murphy ap. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w. Sci Rep. 2025. PMID: 39747998 Free PMC article. Clinical Trial.
Encoding of 3D physical dimensions by face-selective cortical neurons.
Khandhadia AP, Murphy AP, Koyano KW, Esch EM, Leopold DA. Khandhadia AP, et al. Among authors: murphy ap. Proc Natl Acad Sci U S A. 2023 Feb 28;120(9):e2214996120. doi: 10.1073/pnas.2214996120. Epub 2023 Feb 21. Proc Natl Acad Sci U S A. 2023. PMID: 36802419 Free PMC article.
Audiovisual integration in macaque face patch neurons.
Khandhadia AP, Murphy AP, Romanski LM, Bizley JK, Leopold DA. Khandhadia AP, et al. Among authors: murphy ap. Curr Biol. 2021 May 10;31(9):1826-1835.e3. doi: 10.1016/j.cub.2021.01.102. Epub 2021 Feb 25. Curr Biol. 2021. PMID: 33636119 Free PMC article.
46 results