ArticleAnnals of clinical and translational neurology2025
Remaining Burden of Spinal Muscular Atrophy Among Treated Patients: A Survey of Patients and Caregivers.
Article in Annals of clinical and translational neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 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.
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Who cites it
4 citing papers in PubMed.
- Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study.Journal of neurology · 2026Observational
- Adaptive functioning in school-aged children with spinal muscular atrophy in the treatment era: a non-randomised cohort study.The Lancet regional health. Western Pacific · 2026Article
- Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances.Therapeutic advances in neurological disorders · 2026Review
- Behavioral barriers in the management of spinal muscular atrophy: The role of procrastination, regret, and burnout.PloS one · 2026Article
Corrections and comments
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Authors and funding
9 authors.
Funding
Abstract
objectiveSpinal muscular atrophy (SMA) significantly impacts motor function. This study aimed to assess the persistent burden and unmet needs among currently treated patients with SMA and their caregivers.
methodsTwo complementary web-based surveys were distributed in August 2024 among patients with SMA and their caregivers. Non-ambulant patients with SMA currently receiving risdiplam or nusinersen, and/or their primary, informal caregivers were eligible to participate. Survey modules captured clinical, humanistic, productivity, and caregiver-related burden of disease. The PROMIS Fatigue and EQ-5D-5L were used to assess fatigue and quality of life.
results40 pediatric (mean age 8.3 years; represented by caregiver proxies) and 68 adult patients (mean age 37.5 years) were included, of which the majority were on SMN-targeted treatment for ≥ 2 years (82.5% and 94.1%, respectively), and nearly half were on treatment for ≥ 4 years. Despite continued treatment, muscle weakness was reported in 95% of pediatric and 100% of adult patients, with 63% of pediatric and 68% of adult patients reporting "severe" or "very severe" muscle weakness that substantially impacted motor function and performance of activities of daily living. Increased fatigue and muscle weakness were associated with worse overall health. Findings also demonstrated impacts of SMA on patient quality of life and well-being. Most participants reported mobility limitations and muscle weakness as being least improved by current treatment.
interpretationDespite the use of current treatments, there remains a significant burden of SMA on patients and their caregivers. Muscle weakness and mobility limitations remain key areas of unmet need.
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Registered trials
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