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AssigneeFulcrum Therapeutics, Inc.
InventorsAngela Marie Cacace, Luis Gustavo Alejandro Rojas Soto, Lorin A. Thompson, III, Owen Brendan Wallace, Lucienne V. Ronco, Ning Shen, Alan Scott Robertson, Aaron Nakwon Chang
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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, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEF15, or ZNF280A. Methods useful for treating a disease associated with abnormal DUX4 and downstream gene expression (e.g., Fascioscapulohumeral muscular dystrophy) are disclosed.
A method for treating facioscapulohumeral muscular dystrophy (FSHD), the method comprising administering to a subject in need thereof, an effective amount of a p38 kinase inhibitor,
A method of reducing expression levels of a DUX4 polypeptide and/or a polypeptide encoded by a DUX4 downstream gene in muscle cells in a subject in need thereof, the method comprising administering to the subject, an effective amount of a p38 kinase inhibitor selected from:
Dependencies are inferred from the claim text.