Article
A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease.
Genetics in medicine : official journal of the American College of Medical Genetics - 1 Jun 2009
Whybra Catharina, Miebach Elke, Mengel Eugen, Gal Andreas, Baron Karin, Beck Michael, Kampmann Christoph
Abstract excerpt
PURPOSE: Although Fabry disease is X linked and considered to affect primarily male hemizygotes, female heterozygotes may experience all the signs and symptoms of this metabolic disorder. This prospective, single-center, open-label, clinical trial was performed to evaluate the long-term response of female patients with Fabry disease to enzyme replacement therapy. METHODS: Symptomatic women (average age = 47...
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