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Last Updated: March 29, 2024

Claims for Patent: 10,383,864


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Summary for Patent: 10,383,864
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 J. (Emerald Hills, CA), Castelli; Jeff (New Hope, PA)
Assignee: Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:15/974,217
Patent Litigation and PTAB cases: See patent lawsuits and PTAB cases for patent 10,383,864
Patent Claims: 1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient about 50 to about 250 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1, wherein the 1-deoxygalactonojirimycin or salt thereof is administered as an oral dosage form.

3. The method of claim 2, wherein the oral dosage form comprises a tablet, a capsule or a solution.

4. The method of claim 1, wherein the patient does not eat food from about 2 hours before to about 2 hours after administering the 1-deoxygalactonojirimycin or salt thereof.

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

6. The method of claim 1, wherein the patient is male.

7. The method of claim 1, wherein the patient is female.

8. The method of claim 1, wherein the patient is administered about 50 to about 150 mg of the 1-deoxygalactonojirimycin or salt thereof every other day.

9. The method of claim 1, wherein the patient is administered about 150 to about 250 mg of the 1-deoxygalactonojirimycin or salt thereof every other day.

10. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient about 50 to about 250 mg of migalastat hydrochloride every other day.

11. The method of claim 10, wherein the migalastat hydrochloride is administered as an oral dosage form.

12. The method of claim 11, wherein the oral dosage form comprises a tablet, a capsule or a solution.

13. The method of claim 10, wherein the patient does not eat food from about 2 hours before to about 2 hours after administering the migalastat hydrochloride.

14. The method of claim 10, wherein the migalastat hydrochloride enhances .alpha.-galactosidase A activity.

15. The method of claim 10, wherein the patient is male.

16. The method of claim 10, wherein the patient is female.

17. The method of claim 10, wherein the patient is administered about 50 to about 150 mg of the migalastat hydrochloride every other day.

18. The method of claim 10, wherein the patient is administered about 150 to about 250 mg of migalastat hydrochloride every other day.

19. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or a salt thereof every other day, wherein the patient does not eat food from about 2 hours before to about 2 hours after administering the 1-deoxygalactonojirimycin or salt thereof.

20. The method of claim 19, wherein the patient is administered about 150 mg of the 1-deoxygalactonojirimycin or salt thereof every other day.

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