儿童型脊髓性肌萎缩症治疗研究进展

摘要

Spinal muscular atrophy (SMA) is the most common fatal neurogenetic disease in infant period.Clinical manifestations of SMA include symmetrical and progressive weakness and atrophy of proximal limbs.Based on age of incidence,it is divided into child-onset SMA and adult-onset SMA.Childonset SMA has the highest incidence rate.This article summarizes the research progress of SMA therapy in recent years to provide new thoughts for the treatment of SMA.%脊髓性肌萎缩症是婴幼儿期最常见的致死性神经遗传性疾病,临床表现为肢体近端进行性、对称性肌无力和肌萎缩.根据发病年龄可以分为儿童型和成人型,尤以儿童型脊髓性肌萎缩症发病率最高.本文总结近年儿童型脊髓性肌萎缩症的治疗研究进展,旨在为疾病治疗提供新的思路.