Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
申请公布号
CA2699864(C)
申请公布日期
2016.08.02
申请号
CA20082699864
申请日期
2008.09.16
申请人
THE HOSPITAL FOR SICK CHILDREN;MCMASTER UNIVERSITY
发明人
BLANCHARD, JAN E.;BROWN, ERIC D.;TROPAK, MICHAEL B.;MAHURAN, DON J.;BUTTNER, JUSTIN D.
