BENNETT, C. Frank,HUNG, Gene,RIGO, Frank,KRAINER, Adrian R.,HUA, Yimin,PASSINI, Marco A.,SHIHABUDDIN, Lamya,CHENG, Seng H.,KLINGER, Katherine W.
申请号：
DK20170203913T
公开号：
DK3305302(T3)
申请日:
2010.06.17
申请国别(地区):
丹麦
年份:
2018
代理人:
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.
