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Tag Archives: rare/orphan disease therapy

Saving Ryan

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...

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