Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells.

@article{Banugaria2012PersistenceOH,
  title={Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells.},
  author={Suhrad G. Banugaria and Trusha T. Patel and Joanne F Mackey and Stuti Das and Andrea Amalfitano and Amy S. Rosenberg and Joel Charrow and Y T Chen and Priya Kishnani},
  journal={Molecular genetics and metabolism},
  year={2012},
  volume={105 4},
  pages={677-80}
}
With the advent of enzyme replacement therapy (ERT) with alglucosidase alfa (rhGAA, Myozyme®) for Pompe disease, the clinical course of the disease has changed. We have previously described the poor outcome in cross reactive immunologic material (CRIM)-negative and high-titer CRIM-positive (HTCP) patients secondary to high sustained antibody titers (HSAT) which effectively neutralize ERT efficacy. Various immunomodulation strategies are being explored to diminish the immune response to ERT… CONTINUE READING
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Kishnani, Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

  • Y.H.Messinger, N. J. Mendelsohn, +11 authors P.S.A.S. Rosenberg
  • Genetics in medicine: official journal of the…
  • 2012
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