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.: US14054369Application Date: 2013-10-15
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Publication No.: US09095584B2Publication Date: 2015-08-04
- Inventor: Elfrida Benjamin , Hung V. Do , Xiaoyang Wu , John Flanagan , Brandon Wustman
- Applicant: AMICUS THERAPEUTICS
- Applicant Address: US NJ Cranbury
- Assignee: AMICUS THERAPEUTICS, INC.
- Current Assignee: AMICUS THERAPEUTICS, INC.
- Current Assignee Address: US NJ Cranbury
- Agency: Oblon, McClelland, Maier & Neustadt, L.L.P.
- Main IPC: A61K31/445
- IPC: A61K31/445 ; C07K14/00 ; G01N33/68 ; C12Q1/34
Abstract:
The present invention provides methods to determine whether a patient with a lysosomal storage disorder win 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
- US20140206721A1 METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES Public/Granted day:2014-07-24
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