Methods for treatment of Fabry disease
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.
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 α-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 α-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.