Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review.

ABSTRACT:

Objective

Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care. SMA is classified into Types 1-4, with some individuals identified presymptomatically. This systematic review examined the safety and effectiveness of nusinersen and risdiplam for treating SMA.

Methods

We searched research databases, relevant websites and existing systematic reviews. Screening, data extraction and quality assessment were conducted independently by two authors, with discrepancies resolved by a third. Internal quality appraisal ensured methodological rigour. A total of 131 studies reported in 148 sources

SUBMITTER: Mehrabian A 

PROVIDER: S-EPMC12883690 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature