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

· Impostivity Media, LLC
Ebook
352
Pages
Eligible
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About this ebook

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.

About the author

A medical geneticist by training, Dr. Emil Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO of Ultragenyx, a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases. The company has three approved therapies and holds ex-US rights to a fourth approved product, and a deep pipeline of potential treatments across a variety of therapeutic modalities including gene therapy, mRNA therapy, biologics, and small molecule. 

Before founding Ultragenyx in 2010, Dr. Kakkis guided and contributed to the development of multiple approved rare disease products as chief medical officer at BioMarin Pharmaceutical. 

Dr. Kakkis also founded and serves on the Board of EveryLife Foundation for Rare Diseases, a non-profit dedicated to accelerating innovation for rare diseases. 

Dr. Kakkis has been recognized for his leadership by Biotechnology Innovation Organization (BIO), the National MPS Society, WORLDSymposium, World Orphan Drug Congress and California Life Sciences.

In his first book, Saving Ryan, (published June 21,2022) Dr. Kakkis captures the passionate drive and impossible odds faced in order to develop a treatment to save a young boy’s life diagnosed with a disease so rare that no one would ever consider working on it, even though the science existed to treat it.


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