Steven L. Warner,David J. Bearss,Adam Siddiqui-Jain,Clifford J. Whatcott,Jason Marc Foulks,Stephen Patrick Anthony,Paul Flynn,Yuji Fujiwara,Yuka Arikawa
申请号:
US16523829
公开号:
US20200085823A1
申请日:
2019.07.26
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Methods for treating a disease in a subject in need thereof by administering an ACVR1 inhibitor having the following formula (I) are disclosed:including stereoisomers, tautomers, pharmaceutically acceptable salts and prodrugs thereof, wherein R1, R2, R3, and R4 are as defined herein. Subjects that may benefit from treatment may have mutations in their ACVR1 gene. Various diseases may be treated using the described methods, including cancers (e.g., diffuse intrinsic pontine glioma (DIPG)) and genetic disorders (e.g., fibrodysplasia ossificans progressiva (FOP)).