| 发明名称 |
METHOD FOR ENHANCING FOLDING AND TRANSPORT OF MISFOLDED GLUCOCEREBROSIDASE |
| 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 |
US2014249233(A1) |
申请公布日期 |
2014.09.04 |
| 申请号 |
US201414255479 |
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 |
A61K31/137 |
主分类号 |
A61K31/137 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. (canceled) |
| 地址 |
Toronto CA |
