Emil Kakkis's guest appearances

Emil Kakkis

Lectures and Panels

WCG Talks Trials

Tangential Inspiration

National MPS Society: Our Voices

Steve Young

David Fajgenbaum

Scott Hunter

Christopher Austin

Kristin Smedley

Nirav Shah

Andrew Cutler

Leslie Citrome

Susannah Fox

Jeffrey Cooper

Kevin MD

Bryce Olson

Linda Sullivan

Ken Getz

Nathaniel Katz

OwlTail

On this episode: Stephanie interviews Dr. Emil Kakkis, MD, PhD and Ryan Dant about the trials and tribulations of bringing a much needed drug to patients in a community desperately hanging on to the hope of one day having treatment and eventually a cure. Dr. Kakkis is a physician and researcher with a relentless work ethic who embarks on one of the greatest challenges presented to the MPS community; finding a treatment for an incurable disease.  An incurable disease that afflicts children and adults, like Ryan Dant.     Saving Ryan was written by Dr. Kakkis to shine a light on the connection between families struggling to save their children and scientists at the beginning of their careers.  The book has many threads and perspectives, guiding readers through the drug development and FDA approval process.   Dr. Kakkis and Ryan Dant discuss their time together during clinical trials for Aldurazyme and the family-like relationship that they’ve developed.   Ryan speaks about what it was like as a young child in the spotlight and how receiving treatment has altered the course of his life.   Dr. Kakkis not only has an incredible empathy for his patients and the desire to make a difference in their lives, but he is also a leader in the rare disease community.  His experiences with drug development and the manufacturing of Aldurazyme led him to found a pharmaceutical company focused solely on treatment development for rare diseases.  In addition, he is the founder of a non-profit organization dedicated to advocating for legislative changes that will positively impact the lives of all rare disease patients.   To purchase a copy of Saving Ryan, download the latest kindle version from Amazon!  Hardback available soon!Quotes:“What I didn’t appreciate was that the science may not be fancy, but is so powerful if it can change the future of the patients in front of you…if you don’t help anyone, you haven’t really done anything.” --Dr. Emil Kakkis, MD, PhD“When I was younger my parents always told me to not let this disease defeat me, try to live a normal life the best that I can.” --Ryan Dant Mentioned in this episode:AldurazymeThe Ryan Dant Story on 60 MinutesThe Ryan Dant Story in Reader's DigestUltragenyxEveryLife FoundationYou may also be interested in:The Ryan FoundationRaritiesImpositivityThe  National MPS Society exists to cure, support, and advocate for MPS and ML.If you like Our Voices, visit our website and follow us on social media on Facebook, Instagram, Twitter, and LinkedIn. And if you really like the podcast we'd appreciate you telling a friend (maybe even two). 

On this episode:<ul> <li>Stephanie interviews Dr. Emil Kakkis, MD, PhD and Ryan Dant about the trials and tribulations of bringing a much needed drug to patients in a community desperately hanging on to the hope of one day having treatment and eventually a cure.</li> <li>Dr. Kakkis is a physician and researcher with a relentless work ethic who embarks on one of the greatest challenges presented to the MPS community; finding a treatment for an incurable disease.  An incurable disease that afflicts children and adults, like Ryan Dant.   </li> <li> Saving Ryan was written by Dr. Kakkis to shine a light on the connection between families struggling to save their children and scientists at the beginning of their careers.  The book has many threads and perspectives, guiding readers through the drug development and FDA approval process.  </li> <li>Dr. Kakkis and Ryan Dant discuss their time together during clinical trials for Aldurazyme and the family-like relationship that they’ve developed.  </li> <li>Ryan speaks about what it was like as a young child in the spotlight and how receiving treatment has altered the course of his life.  </li> <li>Dr. Kakkis not only has an incredible empathy for his patients and the desire to make a difference in their lives, but he is also a leader in the rare disease community.  His experiences with drug development and the manufacturing of Aldurazyme led him to found a pharmaceutical company focused solely on treatment development for rare diseases.  In addition, he is the founder of a non-profit organization dedicated to advocating for legislative changes that will positively impact the lives of all rare disease patients.  </li> </ul>To purchase a copy of <a href="https://read.amazon.com/kp/embed?asin=B09P9Z1VXC&amp;preview=newtab&amp;linkCode=kpe&amp;ref_=cm_sw_r_kb_dp_4QPYG2N0GWKK976R1GT5" rel="nofollow" target="_blank">Saving Ryan</a>, download the latest kindle version from Amazon!  Hardback available soon!Quotes:“What I didn’t appreciate was that the science may not be fancy, but is so powerful if it can change the future of the patients in front of you…if you don’t help anyone, you haven’t really done anything.” --Dr. Emil Kakkis, MD, PhD “When I was younger my parents always told me to not let this disease defeat me, try to live a normal life the best that I can.” --Ryan Dant Mentioned in this episode: <a href="https://www.aldurazyme.com/patients" rel="nofollow" target="_blank">Aldurazyme</a> <a href="https://youtu.be/0a94lkbXtHg" rel="nofollow" target="_blank">The Ryan Dant Story on 60 Minutes</a> <a href="https://ryanfoundation.org/files/2613/9223/4942/2001ReadersDigest.pdf" rel="nofollow" target="_blank">The Ryan Dant Story in Reader's Digest</a> <a href="https://www.ultragenyx.com/" rel="nofollow" target="_blank">Ultragenyx</a> <a href="https://everylifefoundation.org/" rel="nofollow" target="_blank">EveryLife Foundation</a>You may also be interested in: <a href="https://ryanfoundation.org/home/" rel="nofollow" target="_blank">The Ryan Foundation</a> <a href="https://www.youtube.com/c/Rarities" rel="nofollow" target="_blank">Rarities</a> <a href="https://impositivity.com/" rel="nofollow" target="_blank">Impositivity</a>The  National MPS Society exists to cure, support, and advocate for MPS and ML.If you like Our Voices, visit <a href="https://mpssociety.org/" rel="nofollow" target="_blank">our website</a> and follow us on social media on<a href="https://www.facebook.com/NationalMPSSociety/" rel="nofollow" target="_blank"> Facebook</a>, <a href="https://www.instagram.com/mpssociety/" rel="nofollow" target="_blank">Instagram</a>, <a href="https://twitter.com/MPSSociety" rel="nofollow" target="_blank">Twitter</a>, and <a href="https://www.linkedin.com/company/national-mps-society" rel="nofollow" target="_blank">LinkedIn</a>. And if you really like the podcast we'd appreciate you telling a friend (maybe even two).

Saving Ryan with Dr. Emil Kakkis and Ryan Dant

In this episode, WCG’s President of Patient Advocacy, Steve Smith, talks with Emil Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx Pharmaceuticals and President of the non-profit EveryLife Foundation for Rare Diseases organization. Known for his work in developing an enzyme replacement therapy for the rare disorder MPS I and his connection to the Ryan Foundation, Dr. Kakkis shares with us his experiences in being involved in bringing drugs to market and forming relationships with patients along the way. 

In this episode, WCG’s President of Patient Advocacy, Steve Smith, talks with Emil Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx Pharmaceuticals and President of the non-profit EveryLife Foundation for Rare Diseases organization. Known for his work in developing an enzyme replacement therapy for the rare disorder MPS I and his connection to the Ryan Foundation, Dr. Kakkis shares with us his experiences in being involved in bringing drugs to market and forming relationships with patients along the way.

Developing Novel Treatments for Rare Disorders: A Look at MPS I with Dr. Emil Kakkis

http://www.einstein.yu.edu - Emil Kakkis, M.D., Ph.D., president of Everylife Foundation and Ultragenyx Pharmaceuticals, presents "Improvement of the Development Process for Rare Disease Therapeutics" at the 2013 "Transforming Clinical and Translational Research" symposium hosted by the Harold and Muriel Block Institute for Clinical and Translational Research at Einstein and Montefiore (ICTR).

<a href="http://www.einstein.yu.edu" rel="nofollow" target="_blank">http://www.einstein.yu.edu</a> - Emil Kakkis, M.D., Ph.D., president of Everylife Foundation and Ultragenyx Pharmaceuticals, presents "Improvement of the Development Process for Rare Disease Therapeutics" at the 2013 "Transforming Clinical and Translational Research" symposium hosted by the Harold and Muriel Block Institute for Clinical and Translational Research at Einstein and Montefiore (ICTR).

Translational Medicine Symposium 2013: Dr. Emil Kakkis, 3 of 4