Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.

@article{Banikazemi2007AgalsidasebetaTF,
  title={Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.},
  author={Maryam Banikazemi and Jan Bultas and Christoph Wanner and William R Wilcox and Chester B. Whitley and Marie Sherry McDonald and Richard S. Finkel and Seymour Packman and Daniel Georges Bichet and David G. Warnock and Robert J Desnick},
  journal={Annals of internal medicine},
  year={2007},
  volume={146 2},
  pages={
          77-86
        }
}
Context Fabry disease is an X-linked storage disorder characterized by deficient lysosomal enzyme activity and excessive deposition of glycosphingolipids in vascular endothelial cells. Contribution In this double-blind multicenter trial, 82 adults with kidney dysfunction from Fabry disease were randomly assigned to infusions of enzyme replacement with agalsidase beta or placebo every 2 weeks for up to 35 months. Agalsidase beta reduced the frequency of and delayed the time to clinical events… CONTINUE READING
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