US2022282254A1PendingUtilityA1
P38 kinase inhibitors reduce dux4 and downstream gene expression for the treatment of fshd
Est. expiryOct 5, 2037(~11.2 yrs left)· nominal 20-yr term from priority
Inventors:Angela CacaceLuis Gustavo Alejandro Rojas SotoLorin A. Thompson, IiiOwen Brendan WallaceLucienne V. RoncoNing ShenAlan Scott RobertsonAaron Nakwon Chang
A61K 31/4418A61K 45/00A61P 21/00A61K 31/4439A61K 31/5377A61K 31/53C12Y 207/11024G01N 2500/10C12Q 2600/106C12Q 2600/156A61K 31/437A61K 31/513A61K 31/519A61K 31/496C12Q 1/6883A61K 31/506A61K 31/00A61K 31/454A61K 31/416A61K 9/0053A61K 9/0073C12Q 1/686G01N 33/68C12Q 1/6827A61K 45/06C12N 9/12A61K 9/0019G01N 33/5061C07K 14/4707C12N 15/113
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Claims
Abstract
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.
Claims
exact text as granted — not AI-modifiedWhat is claimed is:
1 . A method of treating facioscapulohumeral muscular dystrophy in a patient in need thereof, comprising orally administering to the patient a therapeutically effective amount of a compound represented by Formula (V′):
or a pharmaceutically acceptable salt thereof.
2 . The method of claim 1 , comprising administering to the patient 15 mg of the compound twice daily.
3 . A method of treating facioscapulohumeral muscular dystrophy in a patient in need thereof, comprising orally administering to the patient, twice daily, a pharmaceutically acceptable tablet comprising a therapeutically effective amount of a compound represented by Formula (V′):
or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient.
4 . A method of treating facioscapulohumeral muscular dystrophy in a patient in need thereof, comprising orally administering to the patient, twice daily, a pharmaceutically acceptable tablet comprising 15 mg of a compound represented by Formula (V′):
and a pharmaceutically acceptable excipient.Cited by (0)
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