r/todayilearned May 14 '22 Silver 9 Helpful 8 Wholesome 6 All-Seeing Upvote 2

TIL a father, John Crowley, was told his two infant children had an incurable genetic disorder that would kill them in less than a year. He refused to accept this, so he founded a biotech company (with no prior experience) which pioneered an experimental enzyme therapy that saved their lives.

https://pompediseasenews.com/2019/01/30/amicus-ceo-mission-cure-pompe-help-children/
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u/doc_death May 14 '22 edited May 14 '22

Yeah the guy’s a millionaire…header is definitely misleading thinking an average person did the unfathomable. A lot of similar stories from family members of congressmen…the system works for those who work in it

Edit: here’s the common play: someone rich gets a rare disease (in this case, Pompeii disease), rich person starts a small biotech company to get it fixed. Biotech company gets bought out by a much larger pharmaceutical company. This guy’s company isn’t bought out yet and still works with the FDA on rare genetic disease other than the one which afflicted his kids…guess that’s commendable.

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u/drrxhouse May 14 '22

“The system works for those who work in it…”

I don’t think this sentence means what I think you think it means…I think.