Key result
Gaining muscle strength (97.3%), improving daily functioning (90.6%), achieving new motor function (89.3%), and stabilizing motor function (88.6%) were the top unmet treatment priorities for adults with SMA.
Why the study?
Despite therapeutic advances for spinal muscular atrophy, unmet needs persist, motivating characterization of unmet needs adults with SMA hope future therapies will address.
Cross-Sectional (n=410)
Adults with SMA prioritize gaining muscle strength and improving daily functioning, with those having more severe disease emphasizing lung and bulbar function.
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Unmet needs in adult SMA patients characterized; hypothesis-generating and requires prospective validation before informing therapy development.
Peterson et al. (2025) conducted a cross-sectional in Spinal muscular atrophy (n=410). Spinal muscular atrophy was evaluated on Most frequently reported unmet needs for future therapies. Gaining muscle strength (97.3%), improving daily functioning (90.6%), achieving new motor function (89.3%), and stabilizing motor function (88.6%) were the top unmet treatment priorities for adults with SMA.
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