Clinical Application of Risdiplam in 5q Spinal Muscular Atrophy: A Narrative Review.

5q spinal muscular atrophy (SMA) is caused by mutations in the survival motor neuron (SMN) gene located on chromosome 5, leading to insufficient SMN protein levels. Risdiplam is an RNA splicing modifier that modifies pre-mRNA splicing of the SMN2 gene, thereby promoting the production of functional survival motor neuron protein (SMN-fl). This article reviews the drug trials of Risdiplam, summarizes the actual clinical data, and systematically evaluates the effectiveness and safety of this drug. By discussing the mechanism of action and economic cost of this drug and comparing it to other SMA drugs, this paper provides a reference for the clinical use of the drug and an idea for future clinical research.