Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review
Risdiplam shows a favorable safety profile and provides both disease stabilization and multidimensional benefits across all functional phenotypes in adults with SMA, although further longitudinal studies using standardized outcome measures are needed to clarify its long-term impact.
Authors
- Paolo Alonge (ORCID: https://orcid.org/0009-0001-3627-8974)
- G Gadaleta (ORCID: https://orcid.org/0000-0001-6563-8222)
- Guido Urbano (ORCID: https://orcid.org/0009-0002-8596-6279)
Institutions
- University of Turin (IT)
- University of Palermo (IT)
Publication Details
- Journal
- Journal of Neurology
- Published
- 2025-12-08
- DOI
- https://doi.org/10.1007/s00415-025-13557-4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Università degli Studi di Torino