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Treatment of Rett Syndrome
专利权人:
Cold Spring Harbor Laboratory
发明人:
Tonks, Nicholas,Krishnan, Navasona
申请号:
AU2020202426
公开号:
AU2020202426A1
申请日:
2020.04.08
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
#$%^&*AU2020202426A120200507.pdf#####The present invention relates to agents and methods for treating autism spectrum disorders, such as Rett Syndrome. This invention addresses the need mentioned above by providing agents and methods for treating or ameliorating symptoms of neurologic diseases, such as autism spectrum disorders and Rett Syndrome. In one aspect, the invention provides a method for treating a neurologic disease associated with one or more mutations in the MECP2 gene. In another aspect the invention provides a method for treating Rett Syndrome, comprising administering to a human subject in need thereof an effective amount of a therapeutic agent that is {[2-bromo-4-(2carbamoyl-2methanesulfonylaminoethyl)phenyl]difluoromethyl}-phosphonic acid (CPT157633). In a further aspect, the invention provides a system comprising a first pharmaceutical composition comprising an effective amount of a first therapeutic agent that is a small molecule inhibitor of PTP1B, and a kit for diagnosing Rett Syndrome, as well as a composition comprising a small molecule inhibitor of PTP1B for use in treatment of a neurologic disorder characterized by mutations in the MECP2 gene.WO 2016/049110 PCTIUS2015/051594 All II
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