[EN] P38 KINASE INHIBITORS REDUCE DUX4 AND DOWNSTREAM GENE EXPRESSION FOR THE TREATMENT OF FSHD<br/>[FR] INHIBITEURS DE LA KINASE P38 RÉDUISANT L'EXPRESSION DU GÈNE DUX4 ET DES GÈNES AVAL POUR LE TRAITEMENT DE LA FSHD
申请人:FULCRUM THERAPEUTICS INC
公开号:WO2019071147A1
公开(公告)日:2019-04-11
The disclosure relates to methods and compositions including p38 kinase inhibitors and agents that regulate expression of DUX4 and downstream genes including but not restricted to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDClL, RFPL2, CCNAl, SLC34A2, TPRXl, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEFl5, or ZNF280A. Methods useful for treating a disease associated with abnormal DUX4 and downstream gene expression (e.g., Fascioscapulohumeral muscular dystrophy) are disclosed.