Status quo of the treatment of spinocerebellar ataxia towards its pathogenesis.
10.3760/cma.j.cn511374-20190707-00339
- VernacularTitle:从发病机制谈脊髓小脑性共济失调的治疗现状
- Author:
Man DING
1
;
Zuneng LU
Author Information
1. Department of Neurology, Renmin Hospital of Wuhan University, Hubei 430060, China. lzn196480@126.com.
- Publication Type:Journal Article
- From:
Chinese Journal of Medical Genetics
2020;37(9):1043-1047
- CountryChina
- Language:Chinese
-
Abstract:
Spinocerebellar ataxia (SCA) is a group of autosomal dominant hereditary diseases. Based on their inheritance pattern, they can be divided into SCAs caused by expansion of microsatellite repeats or point mutations. Although SCAs may be diagnosed based on their clinical characteristics and results of genetic testing, their treatment still remains as a challenge. So far no drug has been approved by the US Food and Drug Administration or the European Medicines Agency. Strict preclinical trials are critical for the development of disease-modifying drugs.
