Research progress on biomarkers for the monitoring of Spinal muscular atrophy.
10.3760/cma.j.cn511374-20221111-00784
- Author:
Yemin JIAN
1
;
Liwen WU
Author Information
1. The School of Pediatrics, Hengyang Medical School, University of South China, Department of Neurology, Hengyang Medical School, University of South China, Hengyang, Hunan 421001, China. 271417152@qq.com.
- Publication Type:Journal Article
- MeSH:
Child;
Humans;
Muscular Atrophy, Spinal/genetics*;
Biomarkers;
Prognosis
- From:
Chinese Journal of Medical Genetics
2024;41(1):106-112
- CountryChina
- Language:Chinese
-
Abstract:
Spinal muscular atrophy (SMA) is the most common neuromuscular disease in children, which seriously affects children's health. At present, gene and molecular modification therapy for SMA have become hot spots. However, there are many uncertainties about when people with SMA should start treatment, how well the drugs can treat, and the prognosis. Therefore, reliable biomarkers for monitoring and evaluation are urgently needed. This review will summarize the progress made in SMA biomarker research in recent years.