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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy.
Zaidman CM, Goedeker NL, Aqul AA, Butterfield RJ, Connolly AM, Crystal RG, Godwin KE, Hor KN, Mathews KD, Proud CM, Kula Smyth E, Veerapandiyan A, Watkins PB, Mendell JR. Zaidman CM, et al. Among authors: proud cm. J Neuromuscul Dis. 2024;11(3):687-699. doi: 10.3233/JND-230185. J Neuromuscul Dis. 2024. PMID: 38607761 Free PMC article.
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
Servais L, Day JW, De Vivo DC, Kirschner J, Mercuri E, Muntoni F, Proud CM, Shieh PB, Tizzano EF, Quijano-Roy S, Desguerre I, Saito K, Faulkner E, Benguerba KM, Raju D, LaMarca N, Sun R, Anderson FA, Finkel RS. Servais L, et al. Among authors: proud cm. J Neuromuscul Dis. 2024;11(2):425-442. doi: 10.3233/JND-230122. J Neuromuscul Dis. 2024. PMID: 38250783 Free PMC article.
371 results