| 发明名称 |
ALLOSTERIC CHAPERONES AND USES THEREOF |
| 摘要 |
The present invention relates to an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) for use in the treatment of a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA), to pharmaceutical composition thereof, to a method for increasing the activity of GAA in a subject and to a method for identifying an allosteric non-inhibitory chaperone for GAA. |
| 申请公布号 |
US2015147309(A1) |
申请公布日期 |
2015.05.28 |
| 申请号 |
US201314405575 |
申请日期 |
2013.06.06 |
| 申请人 |
Fondazione Telethon ;Consiglio Nazionale delle Ricerche |
发明人 |
Parenti Giancarlo;Porto Caterina;Moracci Marco;Ferrara Maria Carmina;Cobucci-Ponzano Beatrice;Andria Generoso |
| 分类号 |
A61K31/4172;A61K38/47;G01N33/573;A61K31/40;A61K31/405;A61K31/195;A61K31/401 |
主分类号 |
A61K31/4172 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
|
| 地址 |
Roma (RM) IT |
