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An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy

OBJECTIVE: The US risdiplam expanded access program (EAP; NCT04256265) was opened to provide individuals with Type 1 or 2 spinal muscular atrophy (SMA) who had no satisfactory treatment options access to risdiplam prior to commercial availability. The program was designed to collect safety data duri...

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Detalles Bibliográficos
Autores principales:	Kwon, Jennifer M., Arya, Kapil, Kuntz, Nancy, Phan, Han C., Sieburg, Cory, Swoboda, Kathryn J., Veerapandiyan, Aravindhan, Assman, Beverly, Bader‐Weder, Silvia, Dickendesher, Travis L., Hansen, Jennifer, Lin, Helen, Yan, Ying, Rao, Vamshi K.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9186129/ https://www.ncbi.nlm.nih.gov/pubmed/35567422 http://dx.doi.org/10.1002/acn3.51560

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