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Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with fabry disease

  • Author(s): Packman, Seymour
  • Germain, DP
  • Charrow, J
  • Desnick, RJ
  • Guffon, N
  • Kempf, J
  • Lachmann, RH
  • Lemay, R
  • Linthorst, GE
  • Ronald, C
  • et al.

Published Web Location

http://europepmc.org/articles/PMC4413801?pdf=render
No data is associated with this publication.
Abstract

Background: Fabry disease results from deficient α-galactosidase A activity and globotriaosylceramide accumulation causing renal insufficiency, strokes, hypertrophic cardiomyopathy and early demise. We assessed the 10-year outcome of recombinant α-galactos

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