SynthesisJournal of neurology2025
Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review.
Synthesis in Journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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
3 citing papers in PubMed.
- Maternal-Fetal Administration of Risdiplam Partially Rescues the SMNΔ7 Mouse Model of Spinal Muscular Atrophy.Annals of neurology · 2026Article
- Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study.Neurology and therapy · 2026Article
- Therapeutic strategies for spinal muscular atrophy: the history and future perspective.Frontiers in human neuroscience · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundRisdiplam has broadened therapeutic options for spinal muscular atrophy (SMA). While its efficacy and safety are well established in children, data in adults remain limited. This review summarizes current evidence on risdiplam use in the adult SMA population.
methodsFollowing PRISMA 2020 guidelines, we systematically reviewed PubMed, Scopus, and the Cochrane Library up to September 2025 for studies including risdiplam-treated adults (≥ 18 years). Outcomes were summarized across motor, bulbar, respiratory, patient-reported, safety, and adherence domains.
resultsFourteen studies (> 200 adults, mainly SMA types 2 and 3) were included. Most participants were non-ambulant and treatment naïve. Motor function was generally stable, with modest yet significant improvements on RULM, HFMSE, or MFM-32, particularly among younger/less severely affected adults. Swallowing, speech, and fatigue often improved even in advanced disease. Patient-reported outcomes consistently indicated perceived gains in quality of life across all phenotypes. Adverse events were mostly mild and transient-mainly gastrointestinal symptoms, photosensitivity, or liver enzyme elevations-with very few temporary discontinuations.
conclusionsRisdiplam 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.