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Last Updated: November 28, 2020

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Claims for Patent: 10,076,514

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Summary for Patent: 10,076,514
Title:Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene
Abstract: Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing .alpha.-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 .alpha.-galactosidase A, wherein the patient has a splice site mutation in intron 4 of the nucleic acid sequence encoding .alpha.-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.
Inventor(s): Benjamin; Elfrida (Millstone Township, NJ)
Assignee: Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:15/459,149
Patent Claims: 1. A method of treating a patient diagnosed with Fabry disease, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or salt thereof, wherein the patient has a mutation in the nucleic acid sequence encoding .alpha.-galactosidase, and wherein the mutation is G9331A relative to SEQ ID NO: 1.

2. The method of claim 1, wherein the dose of 1-deoxygalactonojirimycin or salt thereof is from about 25 mg to about 250 mg.

3. The method of claim 1, wherein the salt of 1-deoxygalactonojirimycin is 1-deoxygalactonojirimycin hydrochloride.

4. The method of claim 1, wherein the dose is about 150 mg every other day of 1-deoxygalactonojirimycin hydrochloride or an equivalent dose of 1-deoxygalactonojirimycin or a salt thereof other than the hydrochloride salt.

5. The method of claim 1, wherein the 1-deoxygalactonojirimycin or salt thereof is administered orally or by injection.

6. A method of treating a human patient diagnosed with Fabry disease, the method comprising orally administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or salt thereof, wherein the patient has a mutation in the nucleic acid sequence encoding .alpha.-galactosidase, wherein the mutation is G9331A relative to SEQ ID NO: 1, and wherein the dose is about 150 mg every other day of 1-deoxygalactonojirimycin hydrochloride, or an equivalent dose of 1-deoxygalactonojirimycin or a salt thereof other than the hydrochloride salt.

7. The method of claim 6, wherein the patient is orally administered about 150 mg of 1-deoxygalactonojirimycin hydrochloride every other day.

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Serving leading biopharmaceutical companies globally:

AstraZeneca
Dow
Medtronic
Colorcon
McKesson
Boehringer Ingelheim

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