FULCRUM THERAPEUTICS INC.;SHEN Ning;CACACE Angela Marie;ROJAS SOTO Luis Gustavo Alejandro;RONCO Lucienne V.;CHANG Aaron Nakwon;THOMPSON Lorin A. III;ROBERTSON Alan Scott;WALLACE Owen Brendan
发明人:
CACACE Angela Marie,ROJAS SOTO Luis Gustavo Alejandro,THOMPSON Lorin A. III,WALLACE Owen Brendan,RONCO Lucienne V.,SHEN Ning,ROBERTSON Alan Scott,CHANG Aaron Nakwon,ROJAS SOTO Luis
申请号:
IL27358920
公开号:
IL273589D0
申请日:
2020.03.25
申请国别(地区):
IL
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).