Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
AssigneeAmicus Therapeutics, Inc.
InventorJeff Castelli
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Abstract
Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
Claims12
A method of reducing left ventricular mass index (LVMi) in an enzyme replacement therapy (ERT)-experienced human patient having Fabry disease, the method comprising administering to the patient a formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof every other day, wherein the effective amount is about 123 mg free base equivalent (FBE) and wherein the patient has left ventricular hypertrophy (LVH) prior to initiating administration of the 1-deoxygalactonojirimycin or salt thereof.
A method of reducing podocyte globotriaosylceramide (GL-3) in an enzyme replacement therapy (ERT)-experienced human patient having Fabry disease, the method comprising administering to the patient a formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof every other day, wherein the effective amount is about 123 mg free base equivalent (FBE).
Dependencies are inferred from the claim text.