Precursor N-acetylgalactosamine-4 sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme

Abstract

The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.

1 - 64 . (canceled) 
     
     
         65 . A method for treating a disease caused all or in part by a deficiency in N-acetylgalactosamine-4-sulfatase activity comprising the step of administering to a human subject in need of such treatment an effective amount of a composition comprising recombinant human N-acetylgalactosamine-4-sulfatase (rhASB). 
     
     
         66 . The method of  claim 65  wherein the amount of rhASB is effective to provide one or more beneficial effects selected from the group consisting of joint mobility, joint pain, joint stiffness, exercise tolerance, exercise endurance, pulmonary function, visual acuity, and activities of daily living. 
     
     
         67 - 68 . (canceled) 
     
     
         69 . The method of  claim 65  wherein the rhASB is administered at a dose of at least 0.2 mg/kg per week. 
     
     
         70 . The method of  claim 69  wherein the rhASB is administered at a dose of at least 1 mg/kg per week. 
     
     
         71 . The method of  claim 65  wherein the rhASB is administered via a once weekly infusion over a period of 2 to 4 hours. 
     
     
         72 . The method of  claim 71  wherein the rhASB is administered via a once weekly infusion over a period of 4 hours. 
     
     
         73 - 74 . (canceled)