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pubmed-article:16950982pubmed:abstractTextFabry disease is an X-linked multisystem disorder. Enzyme-replacement therapy in adults has limited efficacy in treating major sequelae of advanced Fabry disease, such as kidney failure or stroke. This prompted a study of the safety and efficacy of enzyme replacement at an earlier stage of Fabry disease.lld:pubmed
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pubmed-article:16950982pubmed:articleTitleEnzyme-replacement therapy with agalsidase alfa in children with Fabry disease.lld:pubmed
pubmed-article:16950982pubmed:affiliationDevelopmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892-1260, USA.lld:pubmed
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