Claims for Patent: 9,999,618

Summary for Patent: 9,999,618

Title:	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.
Inventor(s):	Castelli; Jeff (New Hope, PA), Lockhart; David J. (Del Mar, CA)
Assignee:	Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:	15/213,920
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 9,999,618
Patent Claims:	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.