Research progress on biomarkers for the monitoring of Spinal muscular atrophy
10.3760/cma.j.cn511374-20221111-00784
- VernacularTitle:脊髓性肌萎缩病情监测生物标志物的研究进展
- Author:
Yemin JIAN
1
;
Liwen WU
Author Information
1. 南华大学衡阳医学院,南华大学儿科学院(湖南省儿童医院)神经内科,衡阳 421001
- Keywords:
Spinal muscular atrophy;
Biomarker;
Surveillance
- 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.