Susan M. Freier,Priyam Singh,Frank Rigo,Paymaan Jafar-nejad,Holly Kordasiewicz
申请号：
US16522133
公开号：
US20200056179A1
申请日:
2019.07.25
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.
