The data shows a significant reduction in mutant huntingtin protein in the cerebrospinal fluid of treated patients.

MSA-C has also been reported to be rarely caused by the gene for another disorder of coordination called spinocerebellar ataxia (SCA3). In the early stages of MSA, levodopa and drugs that act directly ...

7月1日，安天圣施宣布完成近亿元人民币A轮融资。本轮融资由国投创业领投，老股东和瑞创投SCA3产业基金跟投。本轮所募资金将用于支持安天圣施 ...

First clinical data of VO659 demonstrated 28% mean reduction in CSF mutant HTT at day 85 in HD patients in 40 mg dose cohort with an effect after the first dose No sustained ...