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Methods for treating spinal muscular atrophy
专利权人:
Biogen MA Inc.
发明人:
Frank Rigo,Katherine M. Bishop
申请号:
US15509642
公开号:
US10436802B2
申请日:
2015.09.11
申请国别(地区):
US
年份:
2019
代理人:
摘要:
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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