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 IL217014(D0) 申请公布日期 2012.02.29
申请号 IL20110217014 申请日期 2011.12.15
申请人 COLD SPRING HARBOR LABORATORY;ISIS PHARMACEUTICALS, INC.;GENZYME CORPORATION 发明人
分类号 A61K 主分类号 A61K
代理机构 代理人
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