Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AssigneeAmicus Therapeutics, Inc.
InventorsDavid J. Lockhart, Brandon Wustman
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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 treating Gaucher disease comprising orally administering, as part of monotherapy isofagomine treatment, a daily dose of isofagomine of about 75 to 300 mg once daily for about 4 to about 10 days, followed by orally administering a maintenance dose of about 75 to 225 mg once every about 2 to about 8 days.
A method of treating Gaucher disease comprising orally administering, as part of monotherapy isofagomine treatment, isofagomine at a dose of about 75 to 300 mg once every about 2 to about 8 days.
The method 4, wherein the isofagomine is orally administered every 2 to 3 days.
Dependencies are inferred from the claim text.