I just finished reading “Saving Ryan” by Emil Kakkis. Kakkis is a physician who pioneered enzyme replacement therapy for Mucopolysaccharidosis I (MPS I), a progressive and fatal disease. The story is about the patients and their families who struggle with the disease and Emil’s struggle to deliver life-enhancing therapy against terribly long odds. The Ryan of the book’s title is a kid with MPS I. His working class parents were not content to watch Ryan decline and die in his first or second decade. MPS I is an extremely rare disease, so big pharma sees no potential for return on investment for developing treatments or cures. Ryan’s parents start a foundation to raise money for a cure.I seldom read lay science/medical books because they usually spend
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Joel Eissenberg considers the following as important: Featured Stories, Healthcare, MPS I, rare/orphan disease therapy
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I just finished reading “Saving Ryan” by Emil Kakkis. Kakkis is a physician who pioneered enzyme replacement therapy for Mucopolysaccharidosis I (MPS I), a progressive and fatal disease. The story is about the patients and their families who struggle with the disease and Emil’s struggle to deliver life-enhancing therapy against terribly long odds. The Ryan of the book’s title is a kid with MPS I. His working class parents were not content to watch Ryan decline and die in his first or second decade. MPS I is an extremely rare disease, so big pharma sees no potential for return on investment for developing treatments or cures. Ryan’s parents start a foundation to raise money for a cure.
I seldom read lay science/medical books because they usually spend too much time explaining technical details that I already understand, and I find myself wincing at the imprecision and outright errors. This book avoids that pitfall, both by being scientifically sound and concise, and by keeping the focus on the emotionally gripping struggle to validate a therapy and get it approved by FDA regulators and into patients. The patients and their families were essential in generating the initial funding that enabled Kakkis to get proof-of-principle data using a dog model of MPS I. The reader rides an emotional roller coaster as Kakkis works through technical barriers, funding and personnel challenges, corporate critics and regulatory anachronism.
The publisher of “Saving Ryan” is Impositivity Media, which bills itself as “Impossibly Positive Stories.” Emil’s book certainly delivers, although during some of the bleaker sections, I had to remind myself that this has a happy ending. The writing is neither maudlin nor saccharine, but tells the story that connects at a human level.
The mark of a good book is that the reader finds themselves reflecting on what they read long after they put the book down. I’ll be doing that with “Saving Ryan.” I certainly recommend this book, both as a chronicle of clinical translational research and as an uplifting story in our cynical age.