Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy
Risdiplam is overall safe and well-tolerated up to 24 months in adults with SMA. The treatment resulted in improvement or stabilization of motor and respiratory function in non-ambulatory and ambulatory patients. Improvement on the CHOP-ATEND suggests it may be a sensitive marker of change. Longer-term follow-up is needed to understand the impact of risdiplam in adults with SMA.
Authors
- Andrea Jaworek
- Kristina Kelly (ORCID: https://orcid.org/0000-0002-2151-0873)
- Sarah Heintzman
- Matti D. Allen (ORCID: https://orcid.org/0000-0002-0755-3164)
- Gary Sterling
- Kathryn Jira
- Trevor Moravec
- Songzhu Zhao
- W. David Arnold
- Jerold Reynolds
- Marco Tellez
- Stephen J. Kolb
Institutions
- Ottawa Hospital (CA)
- The Ohio State University Wexner Medical Center (US)
- Knox Community Hospital (US)
- Institute for Medical Informatics and Biostatistics (CH)
- Ottawa Hospital Research Institute (CA)
Publication Details
- Journal
- Frontiers in Neurology
- Published
- 2026-01-14
- DOI
- https://doi.org/10.3389/fneur.2025.1694037
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00