The Impact of Nusinersen Treatment on Respiratory Function in Patients with Spinal Muscular Atrophy: A Systematic Review.

ABSTRACT: Background/Objectives: This systematic review evaluated the impact of nusinersen therapy on respiratory health and function in individuals with spinal muscular atrophy (SMA) and determined whether nusinersen improves pulmonary function, focusing on differences based on patient age and the timing of treatment initiation. Methods: A systematic search of PubMed, Ovid Medline, ScienceDirect, and Web of Science databases was conducted up to January 2024 in accordance with the PRISMA guidelines. Thirteen studies were included, comprising clinical trials, observational studies, and case series that focused on respiratory outcomes in SMA patients treated with nusinersen. The data on study design, participant characteristics, nusinersen intervention, respiratory outcomes, and adverse

SUBMITTER: Aldukain M 

PROVIDER: S-EPMC11546051 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature