| 发明名称 |
COMPOSITION AND METHODS FOR MODULATING 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 |
KR20120093138(A) |
申请公布日期 |
2012.08.22 |
| 申请号 |
KR20127001244 |
申请日期 |
2010.06.17 |
| 申请人 |
COLD SPRING HARBOR LABORATORY;ISIS PHARMACEUTICALS, INC. |
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. |
| 分类号 |
A61K48/00;A61K31/7088;A61K31/7105 |
主分类号 |
A61K48/00 |
| 代理机构 |
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