aldurazyme

• Stephanie Cozine introduces Mark Dant as the founder and volunteer executive director of the Ryan Foundation, and former board chair of the Every Life Foundation for Rare Diseases, and former president and CEO of the National NPS Society.
• Mark and his wife founded the Ryan Foundation in 1992 after their son, Ryan, was diagnosed with MPS I.
• The Ryan Foundation has funded millions in research and was instrumental in the development of Aldurazyme, the first and only FDA approved drug for the treatment of MPS I.
• Mark and his family have been key advocates and successfully championed the passing of the Ryan Dan Healthcare Opportunity Act in the US House of Representatives in 2009.
• Mark is a former police officer and retired as an Assistant Chief of Police in 2016, and now spends his time volunteering for the Ryan Foundation and other rare disease nonprofits.
• Stephanie and Mark talk about his experience as a parent of a child with MPS and the journey of advocacy and research.
• Mark explains how times were different when Ryan was diagnosed, with no biotech companies, limited research, and no hope.
• Mark shares how he learned about the disease and did research through library visits and phone calls, and how he eventually found a rare disease conference in Denver.
• Mark remembers a specific moment at the conference when he realized the urgency to change the situation for children with rare diseases.

The Ryan Foundation

The  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 like the podcast, we'd appreciate you telling a friend (maybe even two). 

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:
Aldurazyme
The Ryan Dant Story on 60 Minutes
The Ryan Dant Story in Reader's Digest
Ultragenyx
EveryLife Foundation

You may also be interested in:
The Ryan Foundation
Rarities
Impositivity

The  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).