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"Another promising gene therapy, close to being submitted to the FDA for approval, has been developed by Ohio State University, Nationwide Children's hospital, and Avexis"

I would be very interested to know how the economics of that work out. Is this a classic case of the costs and risks publicized (OSU) and the profits privatized (Avexis) ?

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I believe with so called "Orphan diseases" the economics of research and eventual market release rarely work out for private companies. I believe it takes a research phase funded by charities, grants, and tax breaks for companies to have any interest. As a family affected by this, whatever gets this to market. However, I am sure someone will make money.

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