Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis

Nonadherence to risdiplam was observed in the first year of treatment, especially for patients with SMA type 4 and patients aged 6-12 years. Nonadherence was associated with higher all-cause health care costs, with the most pronounced cost difference for SMA type 2. For adherent patients, those who were highly adherent incurred lower health care costs. These findings underscore the importance of treatment adherence and persistence for patients with SMA receiving risdiplam, particularly for younger children and those with greater disease severity.