COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION

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专利权人:

Ionis Pharmaceuticals; Inc.

发明人:

Susan M. Freier,Holly Kordasiewicz

申请号：

US16344254

公开号：

US20190247420A1

申请日:

2017.11.10

申请国别(地区):

US

年份:

2019

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.

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