Long-term safety and efficacy of enzyme replacement therapy for Fabry disease.

  title={Long-term safety and efficacy of enzyme replacement therapy for Fabry disease.},
  author={William R Wilcox and Maryam Banikazemi and Nathalie Guffon and Stephen Waldek and Philip I Lee and Gabor E. Linthorst and Robert J Desnick and Dominique Paul Germain},
  journal={American journal of human genetics},
  volume={75 1},
Elsewhere, we reported the safety and efficacy results of a multicenter phase 3 trial of recombinant human alpha -galactosidase A (rh-alpha GalA) replacement in patients with Fabry disease. All 58 patients who were enrolled in the 20-wk phase 3 double-blind, randomized, and placebo-controlled study received subsequently 1 mg/kg of rh-alpha GalA (agalsidase beta, Fabrazyme, Genzyme Corporation) biweekly in an ongoing open-label extension study. Evidence of long-term efficacy, even in patients… CONTINUE READING

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a-Galactosidase A deficiency: Fabry disease

  • RJ Desnick, YA Ioannou, CM Eng
  • 2001
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