Observational studyJournal of neurology2026
Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study.
Observational study in Journal of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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
1 citing paper in PubMed.
- Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study.Neurology and therapy · 2026Article
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
BACKGROUND AND
objectiveNusinersen was the first disease-modifying treatment approved for spinal muscular atrophy (SMA). However, long-term results of broad unselected populations-particularly adolescents and adults-remain limited. We aimed to evaluate nusinersen long-term persistence and effectiveness in a population-based cohort.
methodsWe conducted a population-based, ambispective observational study of all SMA patients in the Valencian community (Spain) between 2017 and 2022, with follow-up until December 2025 or censoring (due to death, clinical trial, or treatment switch). Demographic, clinical, and motor outcomes using revised SMA Functional Composite Score (SMA-FCR) were collected. Patients were classified as responders or non-responders. Nusinersen discontinuation risks and motor trajectories were evaluated using Bayesian linear and mixed linear models.
resultsOf 72 patients included, 18 were < 12 years old (all treated with nusinersen) and 54 were ≥ 12 years (28 treated; 26 untreated) at the baseline visit. After a median follow-up of 4.6 years until censoring, all children were found responders, compared with 68% of those ≥ 12 years. Discontinuation rates were 11% in children compared to 75% in the older cohort. In patients ≥ 12 years, reasons for discontinuation included: treatment burden (71%), and loss (53%) or lack of benefit (43%). Lower baseline SMA-FCR (expEstimate = 0.84 [0.718,0.93],prob:1) and older age (expEstimate = 1.028 [1.011,1.055],prob:1) independently predicted higher discontinuation risk. Sustained treatment was associated with SMA-FCR increase, while untreated and discontinued patients showed slight deterioration. DISCUSSION: Nusinersen persistence was high in children but declined significantly after age 12 due to treatment burden and limited efficacy although a 25% of adolescents and younger adults with higher baseline function experienced sustained benefit.
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