Combination therapy for treating protein deficiency disorders

Abstract

This application provides methods of improving protein replacement therapy by combining protein replacement therapy with active site-specific chaperones (ASSC) to increase the stability and efficiency of the protein being administered. The application further provides stable compositions comprising the purified protein and an ASSC, and methods of treatment by administering the compositions.

1 - 20 . (canceled) 
     
     
         21 : A stable pharmaceutical composition formulated for parenteral administration to a human and comprising a purified recombinant human wild-type α-galactosidase A and 1-deoxygalactonojirimycin in a pharmaceutically acceptable carrier. 
     
     
         22 : The stable pharmaceutical composition according to  claim 21 , which is formulated for intravenous, subcutaneous, or intraperitoneal administration. 
     
     
         23 : The stable pharmaceutical composition according to  claim 21 , further comprising:
 a buffer selected from the group consisting of a citrate buffer, a phosphate buffer, an acetate buffer, and a bicarbonate buffer.   
     
     
         24 : A method of treating Fabry disease, the method comprising:
 administering the stable pharmaceutical composition according to  claim 21  to a human patient in need thereof.