Fabry disease: Baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry

@article{Eng2007FabryDB,
  title={Fabry disease: Baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry},
  author={Christine M. Eng and Joanne Fletcher and William R Wilcox and Stephen Waldek and C. Ronald Scott and David O. Sillence and Frank B. Breunig and Joel Charrow and Dominique P Germain and Kathleen Nicholls and Maryam Banikazemi},
  journal={Journal of Inherited Metabolic Disease},
  year={2007},
  volume={30},
  pages={184-192}
}
The Fabry Registry is a global observational research platform established to define outcome data on the natural and treated course of this rare disorder. Participating physicians submit structured longitudinal data to a centralized, confidential database. This report describes the baseline demographic and clinical characteristics of the first 1765 patients (54% males (16% aged < 20 years) and 46% females (13% < 20 years)) enrolled in the Fabry Registry. The median ages at symptom onset and… CONTINUE READING

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