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Last Updated: October 31, 2020

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Claims for Patent: 9,480,682

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Summary for Patent: 9,480,682
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:14/641,707
Patent Claims: 1. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 150 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1, wherein the formulation is administered for at least 2 weeks.

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

4. The method of claim 1, wherein the formulation comprises an oral dosage form.

5. The method of claim 4, wherein the oral dosage form comprises a tablet, a capsule or a solution.

6. The method of claim 1, wherein the formulation comprises an injectable solution.

7. The method of claim 6, wherein the injectable solution comprises a carrier comprising water, an aqueous saline solution, an aqueous dextrose solution or an aqueous glycerol solution.

8. The method of claim 7, wherein the carrier comprises a sterile liquid.

9. The method of claim 1, wherein the 1-deoxygalactonojirimycin or salt thereof enhances .alpha.-galactosidase A activity.

10. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 1-deoxygalactonojirimycin or a salt thereof, wherein the 1-deoxygalactonojirimycin or salt thereof is administered to the patient in an amount of 150 mg every other day for at least 2 weeks.

11. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof is administered orally.

12. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof is administered by injection.

13. The method of claim 10, wherein the salt of 1-deoxygalactonojirimycin comprises migalastat hydrochloride.

14. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof enhances .alpha.-galactosidase A activity.

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