Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

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.

What is claimed is: 
     
         1 . 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). 
     
     
         2 . The method of  claim 1 , wherein the patient has left ventricular hypertrophy (LVH) prior to initiating administration of the 1-deoxygalactonojirimycin or salt thereof. 
     
     
         3 . The method of  claim 1 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity. 
     
     
         4 . The method of  claim 1 , wherein the patient is administered about 123 mg of 1-deoxygalactonojirimycin every other day. 
     
     
         5 . The method of  claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day. 
     
     
         6 . The method of  claim 1 , wherein the formulation comprises an oral dosage form. 
     
     
         7 . The method of  claim 6 , wherein the oral dosage form comprises a tablet, a capsule or a solution. 
     
     
         8 . 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). 
     
     
         9 . The method of  claim 8 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity. 
     
     
         10 . The method of  claim 8 , wherein the patient is administered about 123 mg of 1-deoxygalactonojirimycin every other day. 
     
     
         11 . The method of  claim 8 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day. 
     
     
         12 . The method of  claim 8 , wherein the formulation comprises an oral dosage form. 
     
     
         13 . The method of  claim 12 , wherein the oral dosage form comprises a tablet, a capsule or a solution. 
     
     
         14 . 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). 
     
     
         15 . The method of  claim 14 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity. 
     
     
         16 . The method of  claim 14 , wherein the patient is administered about 123 mg of 1-deoxygalactonojirimycin every other day. 
     
     
         17 . The method of  claim 14 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day. 
     
     
         18 . The method of  claim 14 , wherein the formulation comprises an oral dosage form. 
     
     
         19 . The method of  claim 18 , wherein the oral dosage form comprises a tablet, a capsule or a solution. 
     
     
         20 . The method of  claim 14 , wherein treating Fabry disease comprises one or more of reducing left ventricular mass index (LVMi) or reducing podocyte globotriaosylceramide (GL-3).