SynthesisAdvances in therapy2025
Systematic Review and Meta-analysis of Long-Term Nusinersen Effectiveness in Adolescents and Adults with Spinal Muscular Atrophy.
Synthesis in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
5 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review.Journal of neurology · 2025Pooled it
- Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nature reviews. Neurology · 2026Review
- Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study.Journal of neurology · 2026Observational
- Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.Molecular therapy. Advances · 2026Article
- Advancing personalized spinal muscular atrophy care: matching the right biomarker to the right patient at the right time.Journal of neurology · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionGiven the lifelong progression of spinal muscular atrophy (SMA), understanding the long-term effects of nusinersen treatment is crucial. Prior systematic literature reviews (SLRs) consolidated evidence on the real-world effectiveness of nusinersen in adolescents and adults; however, the publications included in these reviews had a limited follow-up of 10-14 months. As newer publications with longer follow-up and more diverse groups have emerged, we conducted an updated SLR and meta-analysis to evaluate the long-term effectiveness of nusinersen treatment in adolescents and adults across a broad spectrum of SMA.
methodsThe updated SLR included papers published from July 1, 2022 (previous search date) to August 4, 2024 with ≥ 5 individuals ≥ 13 years of age and with ≥ 6 months' data on ≥ 1 selected motor function outcome (Hammersmith Functional Motor Scale-Expanded [HFMSE], Revised Upper Limb Module [RULM], and Six-Minute Walk Test [6MWT]). Changes in motor function after initiating nusinersen were summarized across studies included in the previous and updated SLR. Effect sizes were pooled using random-effects models in the meta-analysis. To understand treatment effects by disease severity, a subgroup meta-analysis by ambulatory status was conducted.
resultsSeventeen publications including 650 individuals from 16 countries followed up to 57 months met the inclusion criteria for the updated SLR. Individuals ranged in age from 13 to 71 years, and approximately 40% were ambulatory. Mean or median improvements or stability in motor function over the follow-up period were consistently reported across 31 studies (including 14 from a previous SLR) over 4 years. In the meta-analysis, statistically significant increases from baseline were observed in all three motor function outcomes. Mean HFMSE improvements were greater among ambulant individuals, while mean RULM improvements were greater among non-ambulant individuals.
conclusionOur findings demonstrate the long-term effectiveness of nusinersen in many adolescents and adults from a diverse SMA population, which could support informed decision-making in clinical practice.
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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.