This story follows John and Aileen Crowley and their three children, the two youngest of whom have a rare "untreatable" genetic disease. Pompe disease gradually degenerates muscle until patients cannot breathe or sit up; it also dangerously enlarges the heart. Determined to try to save his children, John Crowley started up a biotech company to develop an enzyme that would replace the non-functioninging one in his children. Others researchers in other companies were trying different approaches. Everyone made mistakes and created problems along the way.

Finally, in "the mother of all experiments," the four promising drug developments were tested against each other in a double-blind trial. After five years of development, the "special medicine" was finally given to the Crowley children. Daughter Megan, a real fighter and outgoing personality, won over the hearts of the researchers. Interesting conflict of interest issues increase the tension in this race for a cure.


It's hard to put this book down, although the reader can get really frustrated with some of the impulsive and unwise moves John Crowley and others make. On the other hand, his energy and determination to save his children make him unwilling to accept that "nothing could be done" for his kids. The work is full of competitions between biotech companies, research scientists, physicians, parents and health care providers. With time running out, the parents refuse to wait until the research processes and those of the FDA finally work themselves out, but end runs and other ways of bucking the system sometimes backfire. Also the reader understands that only someone with John Crowley's position and connections (Harvard Business School, etc.) could have started companies and raised money for research with such success. It would be out of reach for most Americans.


Harper Collins

Place Published

New York



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