Combination therapies for treatment of spinal muscular atrophy

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专利权人:

The Brigham and Women's Hospital, Inc.;Indiana University Research & Technology Corporation

发明人:

Androphy, Elliot J.,Hodgetts, Kevin,Calder, Alyssa Nicole

申请号：

AU2017223706

公开号：

AU2017223706A1

申请日:

2017.02.23

申请国别(地区):

AU

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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/