Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones
AssigneeAmicus Therapeutics, Inc.
InventorsJeff Castelli, David J. Lockhart
Life of this patent
Estimating the term…
Abstract
The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.
Claims20
A method of reducing left ventricular mass index (LVMi) in a 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).
A method of reducing podocyte globotriaosylceramide (GL-3) in a 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).
A method of treating Fabry disease in a patient in need thereof, 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.