The Brigham and Women's Hospital, Inc.;Indiana University Research and Technology Corporation
发明人:
Elliot J. Androphy,Kevin Hodgetts,Alyssa Nicole Calder
申请号：
US15862751
公开号：
US20180235943A1
申请日:
2018.01.05
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
