US2015352093A1PendingUtilityA1

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

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Assignee: AMICUS THERAPEUTICS INCPriority: Apr 26, 2007Filed: May 15, 2015Published: Dec 10, 2015
Est. expiryApr 26, 2027(~0.7 yrs left)· nominal 20-yr term from priority
Inventors:David J. LockhartBrandon Wustman
A61P 7/06A61P 43/00A61P 9/00A61K 31/45A61K 31/445A61P 25/28A61P 25/16A61P 3/00
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Claims

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.

Claims

exact text as granted — not AI-modified
1 .- 111 . (canceled) 
     
     
         112 . A method of treating Fabry disease in a patient in need thereof, the method comprising administering to the patient from about 50 mg to about 300 mg of 1-deoxygalactonojirimycin or a salt thereof about every 2-3 days. 
     
     
         113 . The method of  claim 112 , wherein from about 125 mg to about 225 mg of 1-deoxygalactonojirimycin or a salt thereof is administered to the patient about every 2-3 days. 
     
     
         114 . The method of  claim 113 , wherein about 150 mg of the 1-deoxygalactonojirimycin or a salt thereof is administered to the patient. 
     
     
         115 . The method of  claim 113 , wherein the 1-deoxygalactonojirimycin or a salt thereof is administered to the patient about every 2 days. 
     
     
         116 . The method of  claim 115 , wherein about 150 mg of the 1-deoxygalactonojirimycin or a salt thereof is administered to the patient. 
     
     
         117 . The method of  claim 113 , wherein the 1-deoxygalactonojirimycin or a salt thereof is 1-deoxygalactonojirimycin hydrochloride. 
     
     
         118 . A method for treating Fabry disease in a patient in need thereof, the method comprising administering to the patient from about 50 mg to about 300 mg of 1-deoxygalactonojirimycin hydrochloride about every 2-3 days. 
     
     
         119 . The method of  claim 118 , wherein about 150 mg of the 1-deoxygalactonojirimycin hydrochloride is administered to the patient. 
     
     
         120 . The method of  claim 118 , wherein the 1-deoxygalactonojirimycin hydrochloride is administered to the patient about every 2 days. 
     
     
         121 . The method of  claim 120 , wherein about 150 mg of the 1-deoxygalactonojirimycin hydrochloride is administered to the patient.

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