Saving Ryan: The 30-Year Journey Into Saving the Life of a Child.

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.

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ספר מוקלט : 1 בנובמבר 2022