[EN] COMPOSITIONS AND METHODS FOR ENHANCING ENZYME ACTIVITY IN GAUCHER, GM1-GANGLIOSIDOSIS/MORQUIO B DISEASE, AND PARKINSON'S DISEASE<br/>[FR] COMPOSITIONS ET PROCÉDÉS PERMETTANT D'AMÉLIORER L'ACTIVITÉ ENZYMATIQUE DANS LA MALADIE DE GAUCHER, LA GANGLIOSIDOSE À GM1, LA MALADIE DE MORQUIO DE TYPE B ET LA MALADIE DE PARKINSON
申请人:HOSPITAL FOR SICK CHILDREN
公开号:WO2009049421A1
公开(公告)日:2009-04-23
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.