| 发明名称 |
COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT. |
| 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. |
| 申请公布号 |
MX2011014004(A) |
申请公布日期 |
2012.06.19 |
| 申请号 |
MX20110014004 |
申请日期 |
2010.06.17 |
| 申请人 |
ISIS PHARMACEUTICALS, INC. |
发明人 |
C. FRANK BENNETT;GENE HUNG;FRANK RIGO;ADRIAN R. KRAINER;YIMIN HUA;MARCO A. PASSINI;LAMYA SHIHABUDDIN;SENG H. CHENG;KATHERINE W. KLINGER |
| 分类号 |
A61K31/7088 |
主分类号 |
A61K31/7088 |
| 代理机构 |
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代理人 |
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| 主权项 |
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