Patents Examined by Karem Cochrane Carlson
  • Method to predict response to pharmacological chaperone treatment of diseases
    Patent number: 10813921
    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: Grant
    Filed: July 26, 2018
    Date of Patent: October 27, 2020
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Elfrida Benjamin, Hung V. Do, Xiaoyang Wu, John Flanagan, Brandon Alan Wustman