CACACE, Angela Marie,ROJAS SOTO, Luis Gustavo Alejandro,THOMPSON III, Lorin A.,WALLACE, Owen Brendan,RONCO, Lucienne V.,SHEN, Ning,ROBERTSON, Alan Scott,CHANG, Aaron Nakwon
申请号:
AU2018346709
公开号:
AU2018346709A1
申请日:
2018.10.05
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
The present invention relates to compositions and methods of inhibiting p38 kinase to reduce gene and protein expression of DUX4 and downstream genes regulated by DUX4. The present invention further relates to methods for treating patients suffering from diseases associated with increased expression of DUX4 or expression of an aberrant form of DUX4, such as Facioscapulohumeral muscular dystrophy (FSHD).