Method of treating Gaucher disease
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.
Claims10
A method for inducing a stable conformation of a misfolded glucocerebrosidase in a patient with Parkinson's Disease, consisting of administering to the patient a pharmaceutically acceptable amount of ambroxol, ambroxol hydrochloride, or bromhexine or a pharmaceutically acceptable salt thereof, wherein said ambroxol, ambroxol hydrochloride, or bromhexine or a pharmaceutically acceptable salt thereof induces a stable conformation of a misfolded glucocerebrosidase.
Dependencies are inferred from the claim text.