Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data.

@article{Mehta2009EnzymeRT,
  title={Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data.},
  author={Amit Mehta and Marcus Beck and Paul Elliott and Roberto Giugliani and Ales Linhart and Gere Sunder-Plassmann and Raphael Schiffmann and Federic Barbey and Markus Ries and Jane R. Clarke},
  journal={Lancet},
  year={2009},
  volume={374 9706},
  pages={1986-96}
}
BACKGROUND We analysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients with Fabry's disease who were enrolled in the Fabry Outcome Survey observational database (FOS). METHODS Baseline and 5-year data were available for up to 181 adults (126 men) in FOS. Serial data for cardiac mass and function, renal function, pain, and quality of life were assessed. Safety and sensitivity analyses were done in patients with baseline and at least one relevant follow-up… CONTINUE READING

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