The U.S.A. as represented by the Secretary; Department of Health and Human Services
发明人:
申请号:
EP12716889.6
公开号:
EP2694047B1
申请日:
2012.04.09
申请国别(地区):
EP
年份:
2018
代理人:
摘要:
The invention provides methods of inhibiting the development or progression of a thioesterase deficiency disorder in a mammal by the administration of a compound that functionally mimics the enzymatic activity of all thioesterases in mammals. Such thioesterase deficiency disorders include cancers and adult- or infant-neuronal ceroid lipofuscinoses (NCLs). The invention also provides small molecule mimics of thioesterases useful in the methods of the invention and pharmaceutical compositions containing the therapeutically effective compounds and methods of using the same.