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.

Read more: https://pompediseasenews.com/2019/01/30/amicus-ceo-mission-cure-pompe-help-children/

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