Invention Grant
- Patent Title: Method to predict response to pharmacological chaperone treatment of diseases
- Patent Title (中): 预测药物分子伴侣治疗疾病反应的方法
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Application No.: US12855468Application Date: 2010-08-12
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Publication No.: US08592362B2Publication Date: 2013-11-26
- Inventor: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
- Applicant: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
- Applicant Address: US NJ Cranbury
- Assignee: Amicus Therapeutics, Inc.
- Current Assignee: Amicus Therapeutics, Inc.
- Current Assignee Address: US NJ Cranbury
- Agency: SorinRand LLP
- Main IPC: C07K14/00
- IPC: C07K14/00

Abstract:
The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
Public/Granted literature
- US20110212996A1 METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES Public/Granted day:2011-09-01
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