COMPOSÉS ET PROCÉDÉS POUR RÉDUIRE L'EXPRESSION D'ATXN3

• 收藏
• 分享

专利权人:

Ionis Pharmaceuticals; Inc.

发明人:

FREIER, Susan, M.,KORDASIEWICZ, Holly

申请号：

EP17869883

公开号：

EP3538656A4

申请日:

2017.11.10

申请国别(地区):

EP

年份:

2020

代理人:

摘要：

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or 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 SCA3.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/