发明授权
- 专利标题: Methods for treating parkinsons disease and parkinsonism
- 专利标题(中): 治疗帕金森病和帕金森综合征的方法
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申请号: US11449528申请日: 2006-06-08
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公开(公告)号: US07964617B2公开(公告)日: 2011-06-21
- 发明人: Brandon Wustman
- 申请人: Brandon Wustman
- 申请人地址: US NJ Cranbury
- 专利权人: Amicus Therapeutics, Inc.
- 当前专利权人: Amicus Therapeutics, Inc.
- 当前专利权人地址: US NJ Cranbury
- 代理机构: Hoffmann & Baron, LLP
- 主分类号: A01N43/40
- IPC分类号: A01N43/40 ; A61K31/445 ; A61K31/435
摘要:
Described is a method for treating an individual having a neurological disorder with an associated mutation or mutations in a gene encoding a lysosomal enzyme. Specifically, the individual is administered a specific pharmacological chaperone for the lysosomal enzyme which increases trafficking of the protein from the ER to the lysosome in neural cells, with or without concomitantly increasing enzyme activity in neural cells. Restoration of trafficking relieves cell stress and other toxicities associated with accumulation of mutant proteins. Restoration of enzyme activity relieves substrate accumulation and pathologies associated with lipid accumulation. In a specific embodiment, the neurological disorder is Parkinson's disease or parkinsonism which is associated with mutations in glucocerebrosidase.
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