Patents Assigned to AMICUS THERAPEUTICS
  • METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
    Publication number: 20170003301
    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 ?-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of ?-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
    Type: Application
    Filed: September 19, 2016
    Publication date: January 5, 2017
    Applicant: AMICUS THERAPEUTICS
    Inventors: Elfrida BENJAMIN, Hung V. Do, Xiaoyang Wu, John Flanagan, Brandon Wustman
  • METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES
    Publication number: 20150342940
    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 ?-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of ?-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
    Type: Application
    Filed: June 5, 2015
    Publication date: December 3, 2015
    Applicant: AMICUS THERAPEUTICS
    Inventors: Elfrida BENJAMIN, Hung V. Do, Xiaoyang Wu, John Flanagan, Brandon Wustman