Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease.

@article{Moore2007EnzymeRT,
  title={Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease.},
  author={David F. Moore and Markus Ries and Evelyn L. Forget and Raphael Schiffmann},
  journal={PharmacoEconomics},
  year={2007},
  volume={25 3},
  pages={
          201-8
        }
}
BACKGROUND Fabry-Anderson disease is an x-linked deficiency of lysosomal alpha-galactosidase A (GALA), resulting in chronic renal failure, cardiac arrhythmia, hypertrophy, valvular disease, pain (acro-paraesthesiae) and stroke, together with premature mortality. The disease has a significant impact on quality of life (QOL), as illustrated by studies using the EQ-5D. A specific treatment is available for Fabry-Anderson disease consisting of intravenous enzyme replacement therapy (ERT) of the… CONTINUE READING
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