Method of treating lysosomal storage disorders
AssigneesThe Hospital for Sick Children, McMaster University
InventorsDon J. Mahuran, Michael B. Tropak, Justin D. Buttner, Jan E. Blanchard, Eric D. Brown
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Abstract
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.
Claims21
(canceled)
A method of treating a lysosomal storage disorder comprising administering to a patient a pharmaceutically acceptable amount of ambroxol, ambroxol derivative or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.