Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
View Patent ↗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.
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 for reducing the patient's LVMi, 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 a-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 for reducing the patient's podocyte GL-3, 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 a-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.