- 专利标题: METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
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申请号: EP15162630.6申请日: 2009-02-12
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公开(公告)号: EP2946785B1公开(公告)日: 2018-10-24
- 发明人: Benjamin, Elfrida , Do, Hung V , Wu, Xiaoyang , Flanagan, John , Wustman, Brandon
- 申请人: Amicus Therapeutics, Inc.
- 申请人地址: 1 Cedar Brook Drive Cranbury, NJ 08512 US
- 专利权人: Amicus Therapeutics, Inc.
- 当前专利权人: Amicus Therapeutics, Inc.
- 当前专利权人地址: 1 Cedar Brook Drive Cranbury, NJ 08512 US
- 代理机构: Miller Sturt Kenyon
- 优先权: US28141P 20080212; US35684P 20080311; US93631P 20080902; US113496P 20081111
- 主分类号: G01N33/68
- IPC分类号: G01N33/68 ; C12Q1/34 ; A61K31/445
摘要:
A method of compiling a treatment reference table that indicates the responsiveness of one or more specific mutations of ±-Gal A to a specific pharmacological chaperone, the method comprising: analysing in vitro the response to the specific pharmacological chaperone in a host cell that has been transformed with a nucleic acid vector that encodes a first mutant ±-Gal A and compiling a table listing responsive and non-responsive mutant forms of ±-Gal A. The specific pharmacological chaperone is 1-deoxygalactonojirimycin or a pharmaceutically salt or ester and the first cell is HEK-293 MSR cell.
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