Advances in real-world research on disease-modifying treatments for spinal muscular atrophy
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.
spinal muscular atrophydisease-modifying therapyefficacysafetyreal-world research
万方数据
维普
