Making Rare Disease Treatments A Priority - John Crowley, Executive Chairman at Amicus Therapeutics

It was on a Friday the 13th in late winter 1997 when John Crowley’s life changed forever.  John and his wife Aileen had been noticing concerning symptoms in their infant daughter Megan for several months, and after a few rounds of testing she was diagnosed with a rare form of muscular dystrophy known as Pompe disease.  Doctors told the Crowleys their daughter likely only had a few years to live, an outlook that ultimately sparked John’s remarkable efforts to find treatments for Megan as well as her younger brother Patrick, who was also struck with Pompe. His family’s amazing journey was the inspiration for the movie Extraordinary Measures starring Brendan Fraser and Harrison Ford.  Join host Shiv Gaglani in this captivating and inspiring conversation with Crowley, now the Executive Chairman at Amicus Therapeutics, to learn about how he and his team are shedding light on some of the rarest diseases in the world, the promise of new technology in genetics, and the need for an Operation Warp Speed to develop rare disease treatments.  As Crowley puts it, “We can beat nature, we think, in the years and decades ahead. We just oftentimes have to beat time.”

Mentioned in this episode: https://amicusrx.com/

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