COMPOSITIONS AND METHODS FOR ENHANCING ENZYME ACTIVITY IN GAUCHER, GM1-GANGLIOSIDOSIS/MORQUIO B DISEASE, AND PARKINSON'S DISEASE

摘要

Therapeutic compositions and methods for treatment of lysosomal storage disorders, such as Gaucher, GM1-gangliosidosis/Morquio B disease and Parkinson's disease are described herein. The compositions comprise compounds having glucocerebrosidase and beta-galactosidase inhibitory activity for use as pharmacological chaperones for mutant forms of the enzyme. Methods of treatment involve providing therapeutically effective amounts of such compounds to subjects in need thereof.