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      Enzyme replacement therapy in Fabry disease: a randomized controlled trial.

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          Abstract

          Fabry disease is a metabolic disorder without a specific treatment, caused by a deficiency of the lysosomal enzyme alpha-galactosidase A (alpha-gal A). Most patients experience debilitating neuropathic pain and premature mortality because of renal failure, cardiovascular disease, or cerebrovascular disease.

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          Author and article information

          Journal
          JAMA
          JAMA
          American Medical Association (AMA)
          0098-7484
          0098-7484
          Jun 06 2001
          : 285
          : 21
          Affiliations
          [1 ] Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, Bldg 10, Room 3D03, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892-1260, USA. rs4e@nih.gov
          Article
          joc10278
          10.1001/jama.285.21.2743
          11386930
          383ecdc3-9746-4bf2-8cd5-bb4e73926725
          History

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