Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Children with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy


The purpose of this research study is to determine the effect(s) of medications that alter the immune system on anti-rhGAA immune response in Pompe patients receiving rhGAA enzyme replacement therapy (ERT), and to understand the immune response in Pompe children treated with ERT that do not make antibodies against ERT. In addition, the study is being conducted to determine whether treating Pompe Disease with medications that affect the immune system has any effect on the overall health or disease progression in patients with Pompe disease.


Children, Anti-rhGAA Immune Response, Pompe Disease, rhGAA Enzyme Replacement Therapy (ERT), MYOZYME

Principal Investigator

Barry J Byrne, M.D.



Contact Information



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