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Last Updated: January 26, 2021

DrugPatentWatch Database Preview

Claims for Patent: 9,000,011

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Summary for Patent: 9,000,011
Title:Methods for treatment of Fabry disease
Abstract: Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.
Inventor(s): Lockhart; David (Del Mar, CA), Castelli; Jeff (New Hope, PA)
Assignee: Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:13/445,338
Patent Claims: 1. A method for treatment of Fabry disease in a patient in need thereof comprising administering to the patient a therapeutically effective dose of 150 mg 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1, wherein 1-deoxygalactonojirimycin or a salt thereof is administered for at least 2 weeks.

3. The method of claim 1, wherein 1-deoxygalactonojirimycin or a salt thereof is administered for at least 6 weeks.

4. The method of claim 1, wherein 1-deoxygalactonojirimycin or a salt thereof is administered for at least 12 weeks.

5. The method of claim 1, wherein the salt of 1-deoxygalactonojirimycin administered is migalastat hydrochloride.

6. The method of claim 1, wherein 1-deoxygalactonojirimycin or a salt thereof is administered orally.

7. A method for enhancing .alpha.-GAL activity in a patient diagnosed with or suspected of having Fabry disease comprising administering to the patient a therapeutically effective dose of 150 mg 1-deoxygalactonojirimycin or a salt thereof every other day.

8. The method of claim 7, wherein 1-deoxygalactonojirimycin or a salt thereof is administered for at least 2 weeks.

9. The method of claim 7, wherein 1deoxygalactonojirimycin or a salt thereof is administered for at least 6 weeks.

10. The method of claim 7, wherein 1-deoxygalactonojirimycin or a salt thereof is administered for at least 12 weeks.

11. The method of claim 7, wherein the salt of 1-deoxygalactonojirimycin administered is migalastat hydrochloride.

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