Two Disabled Dudes

The pre-clinical stage of drug development is largely about safety - answering the question 'will a drug be well tolerated in people?' However, Kristina Bowyer of Ionis Pharmaceuticals helps us understand that the patient voice should be integrated into every stage of the process, including pre-clinical. Plus, Sean loses his focus. Enjoy!

John Crowley is the CEO of Amicus Therapeutics.  He is also a funny and compassionate rare disease Dad.  Join us as we learn life lessons and how to start a company - and Sean learns that a million is quite large.

Amicus Therapeutics: https://www.amicusrx.com/

Extraordinary Measures (John's story), starring Brendan Fraser and Harrison Ford: https://g.co/kgs/u7uFgw

This is the LAUNCH of the Two Disabled Dudes Drug Development Series.  We have 5 experts lined up to field our questions about the process and how we as patients can insert our influence.

This episode is an overview of the Drug Development Process which helps us understand how to avoid spending half a billion dollars on a dead end.

Barbara Tate is the Chief Scientific Officer for the Friedreich's Ataxia Research Alliance (FARA).  She has an impressive resume and a great sense of humor. She went from academic science, to big pharma, to venture capital, to nonprofit, and now she graciously shares her knowledge and wisdom with us.

Our next episode features John Crowley who is played by Brendan Fraser in the movie Extraordinary Measures. 

How do we build an effective Global Rare Disease community?  It all starts with sharing stories and ideas.  Join the dudes as they hear Manish Gore's story about Alport Syndrome, and discuss the differences between the US and India and the Rare Disease World.

A few weeks ago, Sean read an engaging article from the Huffington Post called How to Know if You're an Interrupter or a 'Cooperative Overlapper'.  The discussion of this article lead to an exploration of issues on the endless video conference calls that have become a big part of our lives lately.

Kyle still thinks the term 'Cooperative Overlapper' is just a made up term to make Sean feel better but you be the judge.