COLD SPRING HARBOR LABORATORY;ISIS PHARMACEUTICALS, INC.
发明人:
申请号:
EP12188625.3
公开号:
EP2548560B1
申请日:
2006.06.23
申请国别(地区):
EP
年份:
2015
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.