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If a person is suffering from fabrys disease let's say male age 25-30 what is the cost of treatment through intravenous per year and what is the cost of treatment if he chooses chaperone therapy per year?
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There is no cure for fabry disease. Recombinant alpha-galactosidase a (alpha-gal a), the enzyme that is deficient in patients with fabry disease, and migalastat hydrochloride, an oral pharmacologic chaperone that facilitates trafficking of alpha-gal a to lysosomes, are therapeutic options for eligible individuals. Cost I cannot guide you properly.
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