Fabry's disease cost-effectiveness of therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

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Overview

The cost of the drugs treating Fabry's disease are problematic (approximately $170,000 US a year/patient) and remains a barrier to many patients in some countries. Enzyme replacement therapy (typically infused every two weeks) may be performed in the patient's home by the patients themselves. Enzyme replacement therapy is not a cure, and must be infused recurrently for maximum benefit.