Inside the Orphan Drug Revolution

"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"--.