The Challenge of Developing Drugs for Rare Diseases

John F. Crowley is the Chairman and CEO of Amicus Therapeutics, a publicly traded biotechnology company, which he helped to found in 2005 and is now a 300+ person company in 22 countries. John's involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease -- a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his job and became an entrepreneur as the Co-founder, President, and CEO of Novazyme Pharmaceuticals in 2000, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children's lives). In 2001, Novazyme was acquired by Genzyme Corporation for nearly $200 million. John and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer prize-winning journalist Geeta Anand, The Cure: How a Father Raised $100 Million -- and Bucked the Medical Establishment -- in a Quest to Save His Children. The major motion picture Extraordinary Measures, starring Brendan Fraser and Harrison Ford, is inspired by the Crowley family journey. John is the author of a personal memoir: Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy. John also served as a commissioned intelligence officer in the U.S. Navy Reserve from 2005-2016. He was assigned to the Joint Special Operations Command (JSOC) and is a veteran of the global war on terrorism, with service in Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University of Notre Dame Law School and an M.B.A. from Harvard. He previously served (2014-2016) as the National Chairman of the Make-A-Wish Foundation of America and is a founding board member of the Global Genes Project. John is a Henry Crown Fellow at The Aspen Institute. This is an incredibly powerful episode. Enjoy! Show notes and links for this episode can be found at www.fourhourworkweek.com/podcast. This podcast is brought to you by iD Commerce + Logistics. I'm asked all the time about how to scale businesses quickly. Rule number one: remove unnecessary bottlenecks. Many businesses can do so by outsourcing inventory management and fulfillment to a company that makes this its primary focus. iD Commerce + Logistics is just such a company. It helps online retailers and entrepreneurs outgrow their competition by handling all types of details -- from inventory to packing and shipping. I depended on iD to handle these types of details when I launched The 4-Hour Chef so I could focus on promoting the book. As a listener of this podcast, you can get up to $10,000 off your start-up fees and costs waived by visiting tim.blog/scale or idcomlog.com/tim. This podcast is also brought to you by Wealthfront. Wealthfront is a massively disruptive (in a good way) set-it-and-forget-it investing service, led by technologists from places like Apple and world-famous investors. It has exploded in popularity in the last two years and now has more than $2.5B under management. In fact, some of my good investor friends in Silicon Valley have millions of their own money in Wealthfront. Why? Because you can get services previously limited to the ultra-wealthy and only pay pennies on the dollar for them, and it's all through smarter software instead of retail locations and bloated sales teams. Check out wealthfront.com/tim, take their risk assessment quiz, which only takes 2-5 minutes, and they'll show you -- for free -- exactly the portfolio they'd put you in. If you want to just take their advice and do it yourself, you can. Or, as I would, you can set it and forget it. Well worth a few minutes: wealthfront.com/tim.

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Past guests on The Tim Ferriss Show include Jerry Seinfeld, Hugh Jackman, Dr. Jane Goodall, LeBron James, Kevin Hart, Doris Kearns Goodwin, Jamie Foxx, Matthew McConaughey, Esther Perel, Elizabeth Gilbert, Terry Crews, Sia, Yuval Noah Harari, Malcolm Gladwell, Madeleine Albright, Cheryl Strayed, Jim Collins, Mary Karr, Maria Popova, Sam Harris, Michael Phelps, Bob Iger, Edward Norton, Arnold Schwarzenegger, Neil Strauss, Ken Burns, Maria Sharapova, Marc Andreessen, Neil Gaiman, Neil de Grasse Tyson, Jocko Willink, Daniel Ek, Kelly Slater, Dr. Peter Attia, Seth Godin, Howard Marks, Dr. Brené Brown, Eric Schmidt, Michael Lewis, Joe Gebbia, Michael Pollan, Dr. Jordan Peterson, Vince Vaughn, Brian Koppelman, Ramit Sethi, Dax Shepard, Tony Robbins, Jim Dethmer, Dan Harris, Ray Dalio, Naval Ravikant, Vitalik Buterin, Elizabeth Lesser, Amanda Palmer, Katie Haun, Sir Richard Branson, Chuck Palahniuk, Arianna Huffington, Reid Hoffman, Bill Burr, Whitney Cummings, Rick Rubin, Dr. Vivek Murthy, Darren Aronofsky, and many more.

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