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Wake County Services and Resources for Children Birth-5
     
 

 
 

     
 

JANUARY 2003

 

01 - 01

Enzyme Replacment Therapy for Pompe Disease
This study, sponsored by Genzyme Corporation, is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

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