Recent research on the treatment of spinal muscular atrophy.
10.7499/j.issn.1008-8830.2110041
- Author:
Dong-Ling YANG
1
Author Information
1. School of Public Health and Management, Guangxi University of Chinese Medicine, Nanning 530022, China.
- Publication Type:Review
- Keywords:
Gene therapy;
Spinal muscular atrophy;
Survival motor neuron gene
- MeSH:
Child;
Child, Preschool;
Humans;
Infant;
Muscular Atrophy, Spinal/genetics*
- From:
Chinese Journal of Contemporary Pediatrics
2022;24(2):204-209
- CountryChina
- Language:English
-
Abstract:
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscular weakness and atrophy. SMA, as an inherited disease, is the leading cause of death in infants and young children. Rapid progress has been made in the research field of SMA in recent years, and some related treatment drugs have been successfully approved for marketing. This article reviews the recent research advances in the treatment of SMA.
