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 method of treating a patient with Pompe's disease, comprising: administering to the patient a therapeutically effective amount of human acid alpha glucosidase. 
     
     
         2 . The method of  claim 1 , wherein the patient is administered at least 10 mg/kg body weight per week. 
     
     
         3 . The method of  claim 1 , wherein the patient is administered at least 60 mg/kg body weight per week. 
     
     
         4 . The method of  claim 1 , wherein the patient is administered at least 120 mg/kg body weight per week. 
     
     
         5 - 7 . (canceled) 
     
     
         8 . The method of  claim 1 , wherein the alpha-glucosidase is administered intravenously. 
     
     
         9 . (canceled) 
     
     
         10 . The method of  claim 1 , wherein the patient has infantile Pompe's disease. 
     
     
         11 . The method of  claim 10 , wherein the patient survives to be at least one year old. 
     
     
         12 . The method of  claim 1 , wherein the patient has juvenile Pompe's disease. 
     
     
         13 . The method of  claim 1 , wherein the patient has adult Pompe's disease. 
     
     
         14 . The method of  claim 1 , wherein the alpha-glucosidase is predominantly in a 110 kD form. 
     
     
         15 . The method of  claim 1 , further comprising monitoring a level of human acid alpha glucosidase in the patient. 
     
     
         16 - 26 . (canceled) 
     
     
         27 . A pharmaceutical composition comprising human acid alpha glucosidase, human serum albumin, and a sugar in a physiologically acceptable buffer in sterile form. 
     
     
         28 . The pharmaceutical composition of  claim 27  comprising human acid alpha glucosidase, human serum albumin, and glucose in sodium phosphate buffer. 
     
     
         29 . A pharmaceutical composition comprising alpha glucosidase, mannitol and sucrose in an aqueous solution. 
     
     
         30 . The pharmaceutical composition of  claim 27 , wherein the sugar comprises mannitol and sucrose and the concentration of mannitol is 1-3% w/w of the aqueous solution and the concentration of sucrose is 0.1 to 1% w/w of the aqueous solution. 
     
     
         31 . The pharmaceutical composition of  claim 27 , wherein the concentration of mannitol is 2% w/w and the concentration of sucrose is 0.5% w/w. 
     
     
         32 - 34 . (canceled)