发明名称 METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF ß-GLUCOCEREBROSIDASE
摘要 Provided is a method of increasing the stability of wild-type ß- glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of ß-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for ß- glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding ß-glucocerebrosidase. Further provided are ß-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of ß-glucocerebrosidase in vivo in the central nervous system.
申请公布号 WO2007150064(A3) 申请公布日期 2008.02.21
申请号 WO2007US72016 申请日期 2007.06.25
申请人 AMICUS THERAPEUTICS, INC.;WUSTMAN, BRANDON 发明人 WUSTMAN, BRANDON
分类号 A01N43/40;A61K31/445 主分类号 A01N43/40
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