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Not that I want to stop throwing some shade at Sarepta, but I do want to point out that there certainly has been success, at least as an investor, in treating rare diseases. A number of blood cancer treatments started with rare diseases and expanded (say, mantle cell), and and some companies like Alexion and BioMarin were built on conditions I've never heard of.

If you treat less than 5,000 people a year at 300k, you'd top a billion in sales.... Now looking at Novartis' recent gene therapy drug, the price target is, IIRC, between 0.9-1.2 million ... So the gene therapy market is going to be a windfall for whoever can get to right.
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