GLUCOIMIDAZOLE AND POLYHYDROXYCYCLOHEXENYL AMINE DERIVATIVES TO TREAT GAUCHER DISEASE

摘要

The present invention provides glucoimidazole (GIZ) and polyhydroxycyclohexenyl amine (PHCA) derivatives, methods of making them, and methods of use where the GIZ and PHCA derivatives have a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. More particularly, the present invention further provides a method for treating individuals having Gaucher disease by administering the novel GIZ or PHCA derivatives as "chaperones" for the mutant glucocerebrosidase associated with the disease.