Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study.

ABSTRACT:

Introduction/aims

Risdiplam was the first orally administered drug approved to treat spinal muscular atrophy (SMA). Efficacy in adults is based on short-term observational studies. This longitudinal study aimed to examine risdiplam's efficacy and safety in adults over a long period of follow-up.

Methods

All eligible SMA patients ≥ 16 years old, followed at the Muscular Dystrophy Association Hellas Neuromuscular Diseases Unit between April 2021 and December 2023, were included. We prospectively evaluated motor function, muscle strength, and pulmonary function before and after 6, 12, 18, 24, and 30 months of treatment. Laboratory assessments and patient-reported adverse events were recorded.

Results

Overall, 14 patients (18-57 years, 93% treatment-naive) received risdip

SUBMITTER: Gavriilaki M 

PROVIDER: S-EPMC11799401 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature