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Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy

IntroductionRisdiplam has been shown to be safe, well tolerated, and improves or stabilizes motor function in individuals with SMA, but limited published data exists for adults. The aim of this study was to assess the efficacy, safety, and tolerability of risdiplam treatment for adults with SMA.Meth...

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Principais autores: Andrea Jaworek, Kathryn Jira, Matti Allen, Songzhu Zhao, Kristina Kelly, Trevor Moravec, Marco Tellez, Sarah Heintzman, Jerold Reynolds, Gary Sterling, Stephen J. Kolb, William David Arnold, Bakri Elsheikh
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2026-01-01
Colecção:Frontiers in Neurology
Assuntos:
risdiplam
SMA
adults
outcomes
safety
effect
Acesso em linha:https://www.frontiersin.org/articles/10.3389/fneur.2025.1694037/full
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