ArticleNeurology and therapy2025
SMA Community Priorities for Future Drug Therapies: Cross-Sectional Survey Findings Representing 410 Adults with SMA.
Article in Neurology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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
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Who cites it
2 citing papers in PubMed.
- Access to care for adults living with spinal muscular atrophy in the UK.BMJ neurology open · 2026Article
- Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances.Therapeutic advances in neurological disorders · 2026Review
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionDespite advances in the therapeutic landscape for spinal muscular atrophy (SMA), unmet needs for those with this condition persist. This study seeks to characterize unmet needs that adults with SMA hope future therapies will address and explores associations between reported needs and health status and demographic characteristics.
methodsClose-ended questions from 2021 to 2023 Cure SMA Community Update Survey data were used to assess the importance of needs related to muscle and motor function, lung function and bulbar function, and general functioning. Data was stratified by SMA type and mobility status, and Fisher's exact tests were used to assess for statistically significant differences based on these characteristics. Variations in reported needs were further explored with regressions controlling for sex, education, maximum mobility, and drug treatment status.
resultsThe sample included 410 adults who answered questions on unmet needs. Most had type 2 or type 3 SMA (48% and 45%, respectively). Gaining muscle strength was the most frequently reported unmet need, and followed by improving daily functioning, achieving new motor function, and stabilizing motor function. Stratifications and regressions identified statistically significant differences in treatment needs based on health status and demographic characteristics (p < 0.05). Most notably, people with more severe types of SMA and lower mobility were more likely to report items related to lung function and bulbar function as important.
conclusionThis research highlights treatment priorities for adults with SMA.
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