부산대학교 도서관

This article compares the long-term efficacy and safety of two disease-modifying therapies, risdiplam and nusinersen, for children with Type 1 Spinal Muscular Atrophy (SMA). The study conducted matching-adjusted indirect comparisons using patient-level data from two separate trials. The results suggest that children treated with risdiplam may experience greater improvements in survival, motor function, and time to first serious adverse event compared to those treated with nusinersen. However, it is important to consider that these findings may be influenced by unobserved baseline differences between the groups. Further research and consultation of additional data sources are recommended. [Extracted from the article]