Method of treating Gaucher disease

摘要

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.

主权项

1. A method of reducing the symptoms of Gaucher's Disease in an individual suffering from Gaucher's Disease comprising administering to said individual (i) ambroxol, bromhexine, or a pharmaceutically acceptable salt thereof, and (ii) another drug for the treatment of Gaucher's Disease.