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Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

IMPORTANCE: This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene abeparvovec gene replacement therapy. OBJECTIVE: The primary objective of this ongoing study is to assess safety. The secondary obje...

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Detalles Bibliográficos
Autores principales:	Mendell, Jerry R., Al-Zaidy, Samiah A., Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Reash, Natalie F., Iammarino, Megan A., Church, Kathleen R., Kleyn, Aaron, Meriggioli, Matthew N., Shell, Richard
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Medical Association 2021
Materias:	Original Investigation
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8129901/ https://www.ncbi.nlm.nih.gov/pubmed/33999158 http://dx.doi.org/10.1001/jamaneurol.2021.1272

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