发明名称 METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF BETA -GLUCOCEREBROSIDASE
摘要 <p>Provided is a method of increasing the stability of wild-typeβ-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity ofβ-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone forβ-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encodingβ-glucocerebrosidase. Further provided areβ-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity ofβ-glucocerebrosidase in vivo in the central nervous system.</p>
申请公布号 EP2040548(B1) 申请公布日期 2012.05.02
申请号 EP20070812295 申请日期 2007.06.25
申请人 AMICUS THERAPEUTICS, INC. 发明人 WUSTMAN, BRANDON
分类号 A61K31/45 主分类号 A61K31/45
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