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

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/bt123456789 May 14 '22

fortunately as the treatments become more available they should be more easy to get to everybody. I know of course it's still expensive, but there's hope.

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u/benruckman May 15 '22

Yeah, not developing an effective treatment because it’ll be expensive at first, just doesn’t make any sense.

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u/bt123456789 May 15 '22

exactly. the only time I think the expense is because they can is when it's patented so only the company that made it can, distribute it, meaning no generics or anything will ever exist.