Methods for treatment of Fabry disease
View Patent ↗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 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 α-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 α-galactosidase A activity.