Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease.

@article{Eng2001SafetyAE,
  title={Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease.},
  author={Christine M. Eng and Natalie Guffon and William R Wilcox and Dominique P Germain and Paul Lee and Stephen Waldek and Liron Caplan and Gabor E. Linthorst and Robert J Desnick},
  journal={The New England journal of medicine},
  year={2001},
  volume={345 1},
  pages={
          9-16
        }
}
BACKGROUND Fabry's disease, lysosomal alpha-galactosidase A deficiency, results from the progressive accumulation of globotriaosylceramide and related glycosphingolipids. Affected patients have microvascular disease of the kidneys, heart, and brain. METHODS We evaluated the safety and effectiveness of recombinant alpha-galactosidase A in a multicenter, randomized, placebo-controlled, double-blind study of 58 patients who were treated every 2 weeks for 20 weeks. Thereafter, all patients… CONTINUE READING

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