发明名称 |
METHOD OF TREATING LYSOSOMAL STORAGE DISORDERS |
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. |
申请公布号 |
US2014235713(A1) |
申请公布日期 |
2014.08.21 |
申请号 |
US201414255324 |
申请日期 |
2014.04.17 |
申请人 |
The Hospital for Sick Children ;McMaster University |
发明人 |
Mahuran Don J.;Tropak Michael B.;Buttner Justin D.;Blanchard Jan E.;Brown Eric D. |
分类号 |
A61K31/235;A61K31/167;A61K31/136;A61K31/138 |
主分类号 |
A61K31/235 |
代理机构 |
|
代理人 |
|
主权项 |
1. (canceled) |
地址 |
Toronto CA |