Why the study?
Does nusinersen improve motor function and survival in pediatric and adult populations with 5q spinal muscular atrophy?
Does nusinersen improve motor function and survival in pediatric and adult populations with 5q spinal muscular atrophy?
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.
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May support nusinersen as first disease-modifying option in 5q SMA; leaves open optimal timing and long-term durability.
Kacperczyk et al. (2026) studied this question.
Synapse has enriched one closely related paper. Consider it for comparative context: