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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| 主要な著者: | , , , , , , , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Frontiers Media S.A.
2026-01-01
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| シリーズ: | Frontiers in Neurology |
| 主題: | |
| オンライン･アクセス: | https://www.frontiersin.org/articles/10.3389/fneur.2025.1694037/full |
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