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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 effective remedy. Yet these "orphaned" diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. 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. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled.

Velocity Economics is a solutions focused book geared for politicians seeking an alternative to the failed supply-side economic policies that killed the American Dream. Restoring prosperity requires us to understand that America is a Velocity Economy. From business growth to social responsibility, American politicians must pursue policies that will drive prosperity by embracing the principles of Velocity Economics.Early praise for Velocity Economics: "Our cities, counties, and states are being put under immense pressure by the disproved idea that tax cuts cure everything. From rich to poor, skilled to academic, or rural to urban, our communities are more divided now than at nearly any other point in American history. Velocity Economics helps explain why our communities have grown so far apart, and provides a framework for how our leaders can bring America back together."-- State Senator Matt McCoy"Our communities of color have too often been put at the back of the line, struggling in a system that is rigged against them. Velocity Economics demonstrates that the American Dream can only exist when every American has equal opportunity to achieve economic success, sustainability, and financial security."-- Kameron Middlebrooks, President, NAACP Des Moines Branch "Tax policy impacts every household, and policy makers need an updated view of how tax policy truly works. Every economic advisor who expects to win rural America should read Velocity Economics."-- State Representative Chris Hall"What economic gains rural America has seen in recent years has been reliant on downward-trending costs of capital through labor productivity, innovation, and historically low interest rates. Going forward, economic growth will increasingly be reliant on people and businesses taking advantage of labor, innovation, and capital flow opportunities in an increasingly integrated world economy. Expanding rural broadband capacities is crucial to the local velocity of that flow of capital and building wealth."-- Todd Kielkopf, Co-Founder, Community Broadband Action Network"We will only be able to rebuild our economy once we remember that our workers are an asset, not a liability. Velocity Economics is the message all labor leaders need to hear from the politicians they support."-- Edgar Ortiz, Secretary-Treasurer, AFSCME Council 61 Local 3450"Supply-side policies often ironically promote waste in the pursuit of profits. When it comes to food, allowing food waste leaves communities hungry, parents distracted, and kids desperate. Velocity Economics proves what we all know: food recovery efforts reduce the cost of creating well-nourished communities, which are our most productive communities."-- Aubrey Alvarez, Executive Director, Eat Greater Des Moines "We live in a complex world where simple answers seldom offer sustainable solutions. Success in rural America requires accelerating our focus on regeneration and protection of our natural resources. The supply-side policies currently selling our future for short term gain are a perfect example of a simple answer burdened with lasting and negative unintended consequences."-- Seth Watkins, Heritage Farm Owner, Pinhook Farm

George Beadle was a towering scientific figure whose work from the 1930s to 1960 marked the transition from classical genetics to the molecular era. Among other distinctions, he made the pivotal, Nobel Prize-winning discovery with Edward Tatum that the role of genes is to specify proteins. From 1946 to 1960 he led the Caltech Biology Division, rebuilding it to a powerhouse in molecular biology, and afterwards became a successful President of the University of Chicago. This is the first biography of a giant of genetics, written by two of the field's most distinguished contributors, Paul Berg and Maxine Singer.