SYSNAV

• Analysis of 343 SMA patients treated with onasemnogene abeparvovec (OA) confirms that early treatment, especially before six weeks of age, significantly improves motor outcomes. • The study highlights that pre-symptomatic children achieve motor milestones like sitting, standing, and walking earlier and more frequently than symptomatic children. • Findings indicate that while previous treatment with SMN2 splicing modifiers elevates baseline motor scores, it does not significantly alter disease trajectories post-OA therapy. • Safety data from the cohort showed that OA was generally well-tolerated, with the risk of hepatopathy increasing with age, emphasizing the need for careful monitoring.

Real-World Data Reinforces Early Gene Therapy Benefits in Spinal Muscular Atrophy