Includes bibliographical references and index.

"Orphan is about the struggle to save the lives of children who, because of an unlucky roll of the genetic dice, are born with any one of several thousand rare genetic disorders. Many are burdened with diseases that carry mysterious names, some of which you will read about for the first time in this book, along with compelling stories about the physicians, scientists, and parents who have taken them on. Orphan is more than a book about disease and research-it gives voice to thousands of people who, all too often, have endured terrible illnesses, bravely faced arduous clinical trials, and (sometimes) known victories, almost always in silence. It recounts extraordinary breakthroughs and hopes for the future. Many of the disorders that will end our lives are in some part genetically influenced. We really are all orphans, and this book is for all of us"--

"This book is about the struggle to save the lives of children who, because of a roll of the genetic dice, are born with any one of more than several thousand rare genetic disorders. It recounts the now century long effort of small groups of physicians and scientists to take on some of these genetic diseases. In many cases just a few physician-scientists have made an immense contribution to blazing a path toward new therapies"--