Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.

@article{Germain2007SustainedLR,
  title={Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.},
  author={Dominique Paul Germain and Stephen Waldek and Maryam Banikazemi and David A. Bushinsky and Joel Charrow and Robert J Desnick and Philip I Lee and Thomas K Loew and Anouk C. Vedder and Rekha Abichandani and William R Wilcox and Nathalie Guffon},
  journal={Journal of the American Society of Nephrology : JASN},
  year={2007},
  volume={18 5},
  pages={1547-57}
}
Fabry disease, an inherited deficiency of the lysosomal enzyme alpha-galactosidase A, causes progressive intralysosomal accumulation of globotriaosylceramide (GL-3) and premature death from renal, cardiac, and cerebrovascular manifestations. To determine the long-term safety and efficacy of recombinant human alpha-galactosidase A, an open-label, phase III extension study was conducted, involving 58 patients who had classic Fabry disease and completed a 20-wk, double-blind, randomized, placebo… CONTINUE READING
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2 Excerpts

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