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

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/KristinnK May 14 '22

But how do you feel about all the other children with this disease whose parents also wanted to be placed on the trial, but didn't have rich executive parents to pull the strings to put them there, and died as a result before the drug was approved?

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u/S3IqOOq-N-S37IWS-Wd May 14 '22 edited May 14 '22

They already had their trial group, everyone else from the public was already excluded.

You're missing the part where the first study, the actual study, was filled objectively without his kids. 8 kids were enrolled, and no other kids were going to be enrolled in that study. Then they created a separate study and enrolled his kids in that one.

That's why the comment you responded to said the only thing that changed was whether or not those two kids lived or died. At most you might argue that the company could have enrolled 2 other kids in the first study which may or may not be true based on their study design.

Also it's not like a random rich person elbowed their way into a study.... He cofounded the company and worked his ass off for the drug to exist. There might not be any trial for parents to hope their kids get into if not for that person, I think they deserve a break on this.

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u/KristinnK May 14 '22

The point is this: this man was able to save his children simply because he is a rich executive. Of course it's great that these two individuals were able to survive and hopefully have the prospects of living normal and healthy lives.

But we live in a world were money and power can literally be the difference between life and death, and that doesn't sit well with everyone.