ArticleJournal of neuromuscular diseases2026
Disease-modifying therapies for spinal muscular atrophy: Family experience, ethical considerations, and the role of social determinants of health.
Article in Journal of neuromuscular diseases, 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.
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Who cites it
1 citing paper in PubMed.
- Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.Muscle & nerve · 2026Review
Corrections and comments
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Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesSpinal muscular atrophy is a progressive neuromuscular condition associated with a complex chronic disease course. An in-depth understanding of the ethical issues and social determinants of health impacting the experiences of families and children living with this condition is critical to improving care delivery. We identified the ethical tensions faced by families caring for children with spinal muscular atrophy as well as the influence of social determinants of health in relation to their perception of novel disease-modifying therapies.
designQualitative study including semi-structured interviews with caregivers of children with spinal muscular atrophy who received disease-modifying therapies. Interviews were audio recorded, transcribed verbatim, and coded. Thematic analysis was utilized to identify ethically salient themes.
settingThe Hospital for Sick Children (Toronto, Canada)Results:Fifteen family caregivers of children with spinal muscular atrophy type 1 (n = 5), type 2 (n = 5), and type 3 (n = 5) participated. There were three core themes highlighted including (1) best interests of the child, (2) burden of care and associated moral distress, and (3) parent agency. These experiences were impacted by resources and social determinants of health.
conclusionThis study provides important insights into the ethical tensions and relevant social determinants of health impacting the caregiver experience. Understanding the experiences of diverse families will allow more appropriate resource distribution, better counselling and supports for families facing unique psychosocial challenges and treatment burdens, and overall improved delivery of patient and family-centered care.
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