ISIS PHARMACEUTICALS, INC;COLD SPRING HARBOR LABORATORY
发明人:
BAKER, BRENDA F.,KRAINER, ADRIAN R.,HUA, YIMIN
申请号:
DK12188625
公开号:
DK2548560T3
申请日:
2006.06.23
申请国别(地区):
DK
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.