Don J. Mahuran,Michael B. Tropak,Justin D. Buttner,Jan E. Blanchard,Eric D. Brown
申请号:
US12229445
公开号:
US08124597B2
申请日:
2008.08.22
申请国别(地区):
US
年份:
2012
代理人:
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.