Wake Forest University Health Sciences;Genethon;Children's Medical Center Corporation
发明人:
Martin K. Childers,Alan H. Beggs,Ana Maria Buj Bello
申请号：
US15856653
公开号：
US20180369343A1
申请日:
2017.12.28
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
