Immune response hinders therapy for lysosomal storage diseases

Katherine P Ponder1

  • 1Department of Internal Medicine, Washington University School of Medicine, St. Louis, Missouri 63110, USA. KPONDER@DOM.WUSTL.EDU

Summary

Anti-enzyme antibodies reduce the effectiveness of enzyme replacement therapy (ERT) for mucopolysaccharidosis I (MPS I). Transient immunosuppression can improve enzyme uptake and enhance ERT efficacy in MPS I.

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