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14 The cost of treating a patient having the rare genetic Gaucher's liposome storage disease with genetically engineered enzyme is US$ 145 000 to 400 000 per annum according to severity. Who can and will pay? More such situations will occur.
15 Official recognition of orphan drug status is accorded in the USA (pop 240 million) where the relevant disease affects fewer than 200,000 people; in Japan (pop 121 million) for fewer than 50,000 people.
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