John Crowley does not stop at bad news. His two young children, Megan and Patrick, were babies when they were diagnosed with Pompe disease, a rare and fatal form of muscular dystrophy. Crowley changed his entire life in a race against the clock to find a treatment for his kids and reinvented his career at the same time. It was a breakthrough that inspired a Hollywood film, he tells Becky Quick for CNBC Cures. Since then, Crowley has built two biotech companies focused on rare diseases that were later acquired by larger pharmaceuticals and today leads the industry lobbying group, BIO.

Check out the video playlist of the first season of The Path: https://www.youtube.com/playlist?list=PLVbP054jv0Ko-hAZBSDwZaejelC-FMjc_

Join us in advancing awareness and understanding of rare diseases. Visit CNBC.com/Cures to access more clips, resources, or to sign up for our weekly newsletter.

Learn more about rare disease – and what to do in a diagnosis – at the National Organization for Rare Disorders: https://rarediseases.org/

Follow Becky Quick on X: @BeckyQuick

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Bonus: The Path with Becky Quick: A Father’s Rare Determination 7/2/26

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Bonus: The Path with Becky Quick: A Father’s Rare Determination 7/2/26