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 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 α-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.