Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease.

@article{Lenders2016SerumMediatedIO,
  title={Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease.},
  author={M. Lenders and J. Stypmann and T. Duning and B. Schmitz and S. Brand and E. Brand},
  journal={Journal of the American Society of Nephrology : JASN},
  year={2016},
  volume={27 1},
  pages={
          256-64
        }
}
Fabry disease (FD) is a progressive multisystemic disorder, treatable with recombinant enzyme replacement therapy (agalsidase). However, recent studies suggest an endogenous inhibition of agalsidase in patients with FD, as reported for other lysosomal storage diseases. To assess the clinical consequences of serum-mediated agalsidase inhibition in affected patients, we determined the agalsidase inhibition status of 168 patients (68 male) with FD and compared outcomes of inhibition-positive… Expand
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