EP 1117B - Inside The Orphan Drug Revolution

Rare diseases are also called ‘orphan diseases,’ so named because they were long abandoned (or orphaned) by a profit-driven pharmaceutical industry. So why a book about orphan drugs, which by definition treat only rare diseases? Did they really trigger a revolution important enough to care about? And if so, why is that so little known? In fact, orphan drugs have revolutionized society, for reasons ranging from the deeply personal to the broader cultural and political.

Rare diseases deserve our attention because they’re merciless, causing families often far worse human suffering than common diseases. And unlike many common diseases, few can be prevented or treated by a good diet or exercise. Perhaps most importantly, a rare genetic disease could someday strike any family—and an orphan drug become of utmost urgency to anyone with a child or grandchild, niece or nephew.

A biotechnology executive, Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception. His 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. But Geraghty also uses his exceptional experience and vantage point to look forward, to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow.
EP 1117B - Inside The Orphan Drug Revolution
Featuring:
James Geraghty
James Geraghty has been a director of eight NASDAQ-listed biotech companies and chair of five. He’s worked on orphan drugs for over forty 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. A biotechnology executive, James Geraghty has been a passionate participant in the orphan drug revolution since its inception. His book provides eye-witness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made life-saving progress happen. It also reminds us that, if not nurtured, the revolution could end before its immense promise has been fulfilled.v