The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Sep. 16, 2025
Filed:
Jan. 31, 2022
Applicant:
Amicus Therapeutics, Inc., Philadelphia, PA (US);
Inventor:
Elfrida Benjamin, Millstone Township, NJ (US);
Assignee:
Amicus Therapeutics, Inc., Philadelphia, PA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 31/445 (2006.01); A61K 9/00 (2006.01); A61P 3/00 (2006.01);
U.S. Cl.
CPC ...
A61K 31/445 (2013.01); A61K 9/0019 (2013.01); A61K 9/0053 (2013.01); A61P 3/00 (2018.01);
Abstract
Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for α-galactosidase A, wherein the patient has a splice site mutation in intron 4 of the nucleic acid sequence encoding α-galactosidase A. Also described are uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.