Nusinersen effectiveness and safety in children with type II/III 5q-spinal muscular atrophy: Second interim analysis from a Chinese disease registry - PubMed
2 days ago
- #nusinersen
- #pediatric neurology
- #spinal muscular atrophy
- Second interim analysis of nusinersen effectiveness and safety in Chinese children with type II/III 5q-spinal muscular atrophy.
- References include multi-center studies and retrospective analyses on nusinersen's impact on motor function in pediatric SMA patients.
- Studies highlight different trajectories in motor function improvements, with a focus on upper limb and gross motor functions.
- Evidence supports nusinersen's efficacy over 24 months in type 2 and 3 SMA patients.