Advances in real-world research on disease-modifying treatments for spinal muscular atrophy
Journal Title: Journal of Clinical Pediatrics - Year 2025, Vol 43, Issue 1
Abstract
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that leads to muscle weakness, atrophy, and which can lead to death in severe cases. Recently, therapeutic drugs that can modify SMA have emerged and have significantly improved the clinical symptoms and the quality of life of patients. However, the long-term efficacy and safety of these drugs are not yet established, and various confounding factors affecting drug efficacy need further analysis and study. This article reviews the real-world efficacy and safety studies of drugs for SMA modification drugs, intending to provide some new inspirations and thaughts for the precision and individualized treatment of SMA.
Authors and Affiliations
Xian WU, Yan LIU, Xinzhu LIU, Xiaohui HUANG, Jing MA, A-jing XU, Xiaodong XIN, Wengao JIANG, Jian ZHANG
Keywords
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- EP ID EP760000
- DOI 10.12372/jcp.2025.23e0998
- Views 44
- Downloads 0
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