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Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
J Neuromuscul Dis. 2024;11(2):425-442. doi: 10.3233/JND-230122.
J Neuromuscul Dis. 2024.
PMID: 38250783
Free PMC article.
Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.
Proud CM, Mercuri E, Finkel RS, Kirschner J, De Vivo DC, Muntoni F, Saito K, Tizzano EF, Desguerre I, Quijano-Roy S, Benguerba K, Raju D, Faulkner E, Servais L.
Proud CM, et al.
Ann Clin Transl Neurol. 2023 Nov;10(11):2155-2160. doi: 10.1002/acn3.51889. Epub 2023 Sep 10.
Ann Clin Transl Neurol. 2023.
PMID: 37691296
Free PMC article.
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