COLD SPRING HARBOR LABORATORY;ISIS PHARMACEUTICALS, INC.
发明人:
BAKER, BRENDA, F.,KRAINER, ADRIAN, R.,HUA, YIMIN
申请号：
DK06773838
公开号：
DK1910395T3
申请日:
2006.06.23
申请国别(地区):
DK
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.
