发明名称 |
METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF BETA-GLUCOCEREBROSIDASE |
摘要 |
Provided is a method of increasing the stability of wild-type &bgr;-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of &bgr;-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for &bgr;-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding &bgr;-glucocerebrosidase. Further provided are &bgr;-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of &bgr;-glucocerebrosidase in vivo in the central nervous system.
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申请公布号 |
US2011052613(A1) |
申请公布日期 |
2011.03.03 |
申请号 |
US20100941468 |
申请日期 |
2010.11.08 |
申请人 |
AMICUS THERAPEUTICS, INC. |
发明人 |
WUSTMAN BRANDON ALAN |
分类号 |
A61K39/00;A61K31/192;A61K31/198;A61K31/353;A61K31/439;A61K31/4412;A61K31/65;A61K31/713;A61K38/17;A61P25/00;A61P25/16;A61P25/28;A61P29/00;A61P37/04 |
主分类号 |
A61K39/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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