发明名称 Method for the treatment of neurological disorders by enhancing the activity ofβ-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 includes 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.
申请公布号 US7829579(B2) 申请公布日期 2010.11.09
申请号 US20070768043 申请日期 2007.06.25
申请人 AMICUS THERAPEUTICS, INC. 发明人 WUSTMAN BRANDON ALAN
分类号 A01N43/40;A61K31/445;C07D211/00 主分类号 A01N43/40
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