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Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study
Maria Chovi-Trull1, Nancy C Ñungo-Garzón2,3, Karolina A Aragon-Gawinska2
1Pharmacy Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Neurology and Therapy
|May 6, 2026
Summary
Real-world data show high adherence and persistence with risdiplam, an oral treatment for spinal muscular atrophy (SMA). The study supports the feasibility of this therapy for pediatric and adult SMA patients, demonstrating a favorable safety profile.
Area of Science:
- Neurology
- Genetics
- Pharmacology
Background:
- Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder.
- SMN1 gene variants cause SMA, with SMN2 copy number influencing disease severity.
- Risdiplam, an oral SMN2 splicing modifier, offers a therapeutic option for SMA.
Purpose of the Study:
- To evaluate real-world adherence, persistence, and safety of risdiplam in a Spanish SMA cohort.
- To assess the long-term effectiveness of oral risdiplam therapy.
Main Methods:
- Retrospective observational study of genetically confirmed SMA types 1-3 patients treated with risdiplam.
- Adherence assessed via Proportion of Days Covered (PDC) and Morisky-Green questionnaire.
- Persistence defined as time to discontinuation; safety evaluated through adverse event reporting.
Main Results:
- High adherence observed: median PDC of 100% at 12 months; 66% of patients were adherent per Morisky-Green.
- Favorable short- to mid-term persistence: 92.5% remained on treatment at 12 months, with 80.1% at 36 months.
- Low incidence of treatment-related adverse events (7.5%), with one case of vasculitis leading to discontinuation.
Conclusions:
- Risdiplam demonstrates very high adherence and favorable persistence in a real-world SMA population.
- The oral therapy exhibits a favorable safety profile in both pediatric and adult SMA patients.
- These findings support the feasibility and efficacy of risdiplam as an oral treatment option for SMA.
