ArticleFrontiers in neurology2025
Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy.
Article in Frontiers in neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment.RNA biology · 2026Review
- Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.Molecular therapy. Advances · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Introduction: Risdiplam 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. Methods: We conducted a retrospective chart review on adult patients with 5q-SMA who received risdiplam for a minimum of 6 months, including both treatment naïve and those who switched from nusinersen. Baseline demographic data was collected and outcomes included the Revised Upper Limb Module, Children's Hospital of Philadelphia Adult Test of Neuromuscular Disorders (CHOP-ATEND), six-minute walk test, Hammersmith Functional Motor Scale-Expanded, and forced vital capacity. Assessments were performed at baseline, 6, 12, and 24 months. Self-reported adverse effects were recorded. Linear mixed models were used for analysis. Results: Eighteen patients (mean age 41.11 years) met inclusion criteria. CHOP-ATEND scores increased at 12 (+1.99, Discussion: 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.
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