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Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as "rare" because it afflicts 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 ...

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 ...