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Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase
专利权人:
Inc.;Amicus Therapeutics
发明人:
Brandon Alan Wustman
申请号:
US15225029
公开号:
US10064851B2
申请日:
2016.08.01
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for β-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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