Key result
Nusinersen cuts death or permanent ventilation ~47% vs. sham in infants with spinal muscular atrophy.
Why the study?
Does nusinersen improve motor function and survival in pediatric and adult populations with 5q spinal muscular atrophy?
RCT (n=121)
Double-blind
2:1
Does nusinersen improve motor function and survival in pediatric and adult populations with 5q spinal muscular atrophy?
Effect estimate: HR 0.53 (95% CI 0.32-0.89)
Absolute Event Rate: 39% vs 68%
p-value: p=0.0046
Nusinersen is an effective disease-modifying therapy for 5q SMA that improves motor function and survival, though challenges in accessibility and long-term data remain.
No takes yet. Share an insight, caveat, or question.
May support nusinersen as first disease-modifying option in 5q SMA; leaves open optimal timing and long-term durability.
Kacperczyk et al. (2026) conducted an RCT in Spinal muscular atrophy (SMA) (n=121). Nusinersen (Spinraza) vs. Sham procedure was evaluated on Time to death or permanent ventilation (event-free survival) (HR 0.53, 95% CI 0.32-0.89, p=0.0046). Nusinersen reduced the risk of death or permanent ventilation by 47% (HR 0.53) compared to a sham procedure in infants with symptomatic spinal muscular atrophy.
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