Article
Pediatric Fabry disease.
Pediatrics - 1 Mar 2005
Ries Markus, Gupta Surya, Moore David F, Sachdev Vandana, Quirk Jane M, Murray Gary J, Rosing Douglas R, Robinson Chevalia, Schaefer Ellen, Gal Andreas, Dambrosia James M, Garman Scott C, Brady Roscoe O, Schiffmann Raphael
Abstract excerpt
BACKGROUND: Fabry disease is an underdiagnosed, treatable, X-linked, multisystem disorder. OBJECTIVES: To test the hypothesis that quality of life and sweating are decreased among pediatric patients with Fabry disease, compared with control subjects, and to provide quantitative natural history data and novel clinical end points for therapeutic trials. DESIGN: Prospective, cross-sectional, observational study....
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