Summary

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• Nusinersen (Spinraza) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
• The information was derived from 10 systematic reviews/meta-analyses studies.
• Compared to risdiplam, nusinersen significantly increased Hammersmith Functional Motor Scale-Expanded (HFMSE), Revised Upper Limb Module (RULM), and Hammersmith Infant Neurological Evaluation Section 2 (HINE-2) scores with no significant difference in adverse events between the two drugs.
• In children, nusinersen showed an overall adverse event rate of 83.51% with fever and respiratory infections being most common; it had improved safety compared to placebo with lower serious adverse event rates.
• Long-term data indicate that nusinersen improves motor functions across all SMA types, particularly Type 1 patients, showing stability or minor improvements over time with rare adverse events reported.
• For adults older than 12 years with SMA type linked to chromosome 5q-SMA, nusinersen demonstrated clear benefits in motor function improvements while maintaining a low severe adverse event rate (~2%) and a treatment withdrawal rate of ~3%.