Methods for treatment of Fabry disease
AssigneeAmicus Therapeutics, Inc.
InventorsDavid 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.
Claims11
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.
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.
Dependencies are inferred from the claim text.