- 专利标题： COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT
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申请号： EP18185820.0申请日： 2010-06-17
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公开(公告)号： EP3449926A1公开(公告)日： 2019-03-06
- 发明人: BENNETT, C. Frank , HUNG, Gene , RIGO, Frank , KRAINER, Adrian R. , HUA, Yimin , PASSINI, Marco A. , SHIHABUDDIN, Lamya , CHENG, Seng H. , KLINGER, Katherine W.
- 申请人： Biogen MA Inc. , COLD SPRING HARBOR LABORATORY
- 申请人地址： 225 Binney Street Cambridge, MA 02142 US
- 专利权人： Biogen MA Inc.,COLD SPRING HARBOR LABORATORY
- 当前专利权人： Biogen MA Inc.,COLD SPRING HARBOR LABORATORY
- 当前专利权人地址： 225 Binney Street Cambridge, MA 02142 US
- 代理机构： HGF Limited
- 优先权： US21803109P 20090617
- 主分类号： A61K31/7088
- IPC分类号： A61K31/7088 ; C12N15/113
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.
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