Treatment of Pompe's Disease

Abstract

The invention provides methods of treating Pompe's disease using human acid alpha glucosidase. A preferred treatment regime comprises administering greater than 10 mg/kg body weight per week to a patient.

1 . A pharmaceutical composition comprising human acid alpha glucosidase and a simple carbohydrate in a physiologically acceptable buffer in sterile form. 
     
     
         2 . The pharmaceutical composition of  claim 1 , wherein the simple carbohydrate is mannitol and the buffer is a sodium phosphate buffer. 
     
     
         3 . The pharmaceutical composition of  claim 2 , wherein the concentration of mannitol is 1-3% w/w of the solution. 
     
     
         4 . The pharmaceutical composition of  claim 2 , wherein the concentration of mannitol is 2% w/w of the solution. 
     
     
         5 . The pharmaceutical composition of  claim 1 , further comprising a surfactant. 
     
     
         6 . The pharmaceutical composition of  claim 5 , wherein the surfactant is polysorbate 80. 
     
     
         7 . A pharmaceutical composition comprising alpha glucosidase and mannitol in an aqueous solution. 
     
     
         8 . The pharmaceutical composition of  claim 7 , wherein the human acid alpha-glucosidase is at 5 mg/ml and the mannitol is at 2 mg/ml 
     
     
         9 . A lyophilized composition produced by lyophilizing the pharmaceutical composition according  claim 1 . 
     
     
         10 . The lyophilized composition of  claim 9 , further comprising a surfactant. 
     
     
         11 . The lyophilized composition of  claim 10 , wherein the surfactant is polysorbate 80. 
     
     
         12 . A pharmaceutical composition prepared by reconstituting the lyophilized composition of  claim 11  in sterile water. 
     
     
         13 . A lyophilized composition produced by lyophilizing a pharmaceutical composition comprising human acid glucosidase and a bulling agent. 
     
     
         14 . The lyophilized composition of composition of  claim 13  wherein the bulling agent is mannitol. 
     
     
         15 . The lyophilized composition of  claim 14 , further comprising a physiologically acceptable buffer. 
     
     
         16 . The lyophilized composition of  claim 15 , wherein the buffer is a sodium phosphate buffer. 
     
     
         17 . The lyophilized composition of  claim 13 , further comprising a surfactant. 
     
     
         18 . The lyophilized composition of  claim 17 , wherein the surfactant is polysorbate 80. 
     
     
         19 . A pharmaceutical composition prepared by reconstituting the lyophilized composition of  claim 13  in sterile water.