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US 12,599,595 B2Granted
Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AssigneeAmicus Therapeutics, Inc.
InventorsJeff Castelli, David J. Lockhart
Life of this patent
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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.
Claims7
A method of reducing globotriaosylceramide (GL-3) inclusion volume per podocyte in a patient having Fabry disease, the method comprising:
- measuring the GL-3 inclusion volume per podocyte in the patient at baseline prior to administering to the patient a formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof;
- administering to the patient a formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof every other day for at least 6 months, wherein the effective amount is about 123 mg free base equivalent (FBE);
- and
- measuring the GL-3 inclusion volume per podocyte in the patient after 6 months of administering the formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof, wherein the GL-3 inclusion volume per podocyte is reduced after 6 months of administering the formulation comprising an effective amount of 1-deoxygalactonojirimycin or salt thereof.
1 more element
Dependencies are inferred from the claim text.