There are thousands of diseases classed as “rare” because they afflict only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Forty years ago, a revolution started that transformed the prospects of patients with rare diseases. This book is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen, and it tells deeply personal stories of patients and parents willing to risk new, untried therapies.
A widely recognized leader in the rare disease field, Jim Geraghty has been a director or chair of seven NASDAQ-listed biotech companies. He's worked on orphan drugs for more than 40 years―as a strategy consultant, a CEO, a leader of pioneering international operations at Genzyme, and a venture entrepreneur. A former trustee of Harvard Medical School's renowned Joslin Diabetes Center, he's spoken before both houses of Congress, at the World Economic Forum, and at many other high-profile conferences. A Georgetown graduate with a masters in psychology from Penn and a law degree from Yale, he is a citizen of three countries and lives in Boston.