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SmartPeople: Mission possible

Developing medicines for rare diseases requires an entirely different mindset, says John Crowley, chairman and CEO of rare disease company Amicus Therapeutics. “Developing drugs in rare diseases is very different from more prevalent disorders – the clinical studies are very small, you have different endpoints and biomarkers, different regulatory paradigms and different types of sales and marketing, plus the focus on the patient is intense. It takes a fundamentally different mindset than the one in bigger pharma companies.”

For Crowley, it’s all about following the science. “We describe Amicus as business led but science driv-en, and I have always believed that if you pursue great science and make a great medicine that trans-forms people’s lives then the profits will follow.”

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