Compositions and methods for treating spinocerebellar ataxia

Abstract

The present invention provides alleviating a sign or symptom of spinocerebellar ataxia by administering an injection of an aqueous formulation comprising trehalose.

What is claimed is: 
     
         1 . A method of alleviating a sign or symptom of spinocerebellar ataxia (SCA) by administering to a subject in need thereof an injection of an aqueous formulation comprising a single active ingredient consisting of trehalose, wherein the formulation (i) has a pH about 4.5 to 7.0, (ii) contains less than 0.75 endotoxin units per mL, and (iii) is administered at a per administration dose of between 5 to 50 grams trehalose. 
     
     
         2 . The method of  claim 1 , wherein the formulation is administered once weekly. 
     
     
         3 . The method of  claim 1 , where the formulation is administered at a per administration dose of about 0.5 gram trehalose per kilogram body weight. 
     
     
         4 . The method of  claim 1 , wherein the per day dose is 8, 15 or 30 grams. 
     
     
         5 . The method of  claim 1 , wherein the rate of administration is such that the maximum endotoxin level is less than 5 EU per kilogram of body weight per hour. 
     
     
         6 . The method of  claim 1 , wherein the trehalose is at 10% (w/v). 
     
     
         7 . The method of  claim 1 , wherein the formulation has an osmolality of about 280-330 mOsm/kg.