Saving Ryan: The 30 Year Journey Into Saving The Life Of A Child

by Emil D. Kakkis

Saving Ryan is the inspiring story by physician-scientist Dr. Emil Kakkis about his journey to develop a new, first-ever treatment for an ultra-rare genetic disease called MPS. The scientist had great challenges getting grant and industry support for this rare disease despite the solid science behind doing the treatment. Simultaneously across the country, Mark Dant was in a quest to save his 9-year-old son, Ryan, afflicted by MPS for which there was no cure. Through extraordinary persistence, and with a clock-ticking on Ryan’s declining health, Mr. Dant eventually learns of Dr. Kakkis and his work, whose enzyme replacement therapy could potentially treat Ryan successfully. With renewed hope of saving his son’s life, Mr. Dant turns his attention to fundraising through his own Ryan Foundation to support the continued development of the life-saving treatment. Through an extraordinary series of obstacles and heartaches, Dr. Kakkis develops a successful treatment and ultimately gains the required support of a biotech company to complete the project. Despite early success treating MPS patients, the FDA’s abruptly changing policies caused great uncertainty as to whether the therapy would ever get approved – leading to a dramatic showdown at an FDA Advisory Committee meeting. In the end, the treatment was finally approved for all MPS patients, and ultimately saved Ryan. Ryan has been on the enzyme replacement therapy for more than 23 years. He went on to graduate college and in 2021 was married.

488 pages, Kindle Edition

Published January 1, 2022

Reviews

[Dan Morris · Rating 5 out of 5]

I read this because of my buddy (and many peoples buddy!) Bryant Graber that works at the Harris Y. He and his Mom were featured in it as part of the original 10 for the clinical trial of the enzyme therapy. It really helped me to understand more all that he has been through. And to learn about the long process for developing treatments and cures for rare diseases. Dr K outlined how much research, collaboration, and persistence is required to get through trials and approval. An inspiring story, and I would recommend for anyone to read!

[Marta Weber · Rating 5 out of 5]

I am blessed to be an employee at Ultragenyx and work for Dr. Emil Kakkis! I am witness everyday to the fight he leads for every single patient and it’s even more inspiring getting to read about how much he had to overcome to reach approval of his first treatment! Thank God he never gave up!! 💪🏻

[Eduardo O Thompson · Rating 5 out of 5]

This is amazing book is an example of how one’s determination can accomplish any dreams. Dr.Kakkis and the MPS I community have faced so many challenges and hurdles but have never given up.
It is difficult to imagine how much Dr.Kakkis and MPS I patients and families have suffered along the way.
Thank you for sharing your journey and thank you for dedicating your career to helping rare disease patients!