An estimated 25 million people in the US alone collectively live with some sort of orphan disease.
FORBES: Orphan Drugs: 'Rare' Opportunities To Make Money
One reason to purse the unusual use is it is a so-called orphan disease afflicting only about 65, 000 Americans.
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Cumulatively, these orphan disease afflcit 25 million to 30 million people.
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An earlier version of the drug, Ceredase, helped launch the lucrative market for high-priced orphan disease drugs, which is now one of the brightest spots in biotech.
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Which was the top-selling orphan disease drug?
AZT, began as an orphan drug for what was then an obscure disease of the immune system.
ECONOMIST: Rare diseases: Safety in numbers | The
But an alliance of 100 rare-disease support groups from 12 European countries has coalesced around orphan-drug legislation to form Eurordis.
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