COLD SPRING HARBOR LABORATORY;Isis Pharmaceuticals, Inc.
发明人:
申请号:
EP13165321.4
公开号:
EP2644700A1
申请日:
2006.06.23
申请国别(地区):
EP
年份:
2013
代理人:
摘要:
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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.