Methods for treatment of Fabry disease
AssigneeAmicus Therapeutics, Inc.
InventorsDavid J. Lockhart, Jeff Castelli
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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.
Claims20
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.
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.
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.
Dependencies are inferred from the claim text.