Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials.

ABSTRACT:

Objective

We performed a systematic review and meta-analysis of the efficacy and safety of nusinersen and risdiplam in the treatment of spinal muscular disease (SMA).

Methods

We screened the literature published in Pubmed, Web of Science, Embase, and Cochrane before July 2023 to conduct randomized controlled trials to test the treatment of SMA patients with nusinersen and risdiplam. The data were analyzed using Review Manager 5.4 software and Stata version 15.0 software.

Results

A total of six randomized controlled trials were included, involving 728 SMA patients, to synthesize evidence. It is reported that nusinersen treatment was beneficial for increasing the score of the Hammersmith Functional Motor Scale-Expanded (HFMSE) (WMD: 4.90; 95% CI: 3.17, 6.63; p <

SUBMITTER: Qiao Y 

PROVIDER: S-EPMC10605531 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature