Invention Grant
US09402837B2 Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase
有权
通过增强β-葡萄糖脑苷脂酶的活性来治疗神经障碍的方法
- Patent Title: Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase
- Patent Title (中): 通过增强β-葡萄糖脑苷脂酶的活性来治疗神经障碍的方法
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Application No.: US14469008Application Date: 2014-08-26
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Publication No.: US09402837B2Publication Date: 2016-08-02
- Inventor: Brandon Alan Wustman
- Applicant: Amicus Therapeutics, Inc.
- Applicant Address: US NJ Cranbury
- Assignee: Amicus Therapeutics, Inc.
- Current Assignee: Amicus Therapeutics, Inc.
- Current Assignee Address: US NJ Cranbury
- Agency: Servilla Whitney LLC
- Main IPC: A61K31/445
- IPC: A61K31/445 ; A61K31/437 ; A61K31/45 ; A61K31/194 ; A61K31/713

Abstract:
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.
Public/Granted literature
- US20150025109A1 Method for the Treatment of Neurological Disorders by Enhancing the Activity of Beta-Glucocerebrosidase Public/Granted day:2015-01-22
Information query
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