NewsLANCET NEUROLOGYTuesday, September 30, 2025 · September 30, 2025
Zolgensma Long-Term Data: 5-Year Outcomes in SMA
WHY IT MATTERS
Confirms one-time gene therapy provides durable benefit in SMA.
Five-year follow-up of Zolgensma in SMA type 1 shows patients maintaining motor milestones with no new safety signals.