SMA Community Priorities for Future Drug Therapies: Cross-Sectional Survey Findings Representing 410 Adults with SMA.

INTRODUCTION

Despite 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.

METHODS

Close-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.

RESULTS

The 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.

CONCLUSION

This research highlights treatment priorities for adults with SMA.