Inside the Orphan Drug Revolution
The Promise of Patient-Centered Biotechnology
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Narrated by:
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Scott Wallace
About this listen
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 untried new 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.
©2022 James A. Geraghty (P)2022 Made For SuccessListeners also enjoyed...
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New Yorker staff writer Michael Specter has twice won the Global Health Council’s Excellence in Media Award, as well as the Science Journalism Award from the American Association for the Advancement of Science. In Denialism, he fervently argues that people are turning away from new technologies and engaging in a kind of magical thinking that is hindering scientific progress.
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A compelling read
- By S on 05-17-11
By: Michael Specter
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A Crack in Creation
- Gene Editing and the Unthinkable Power to Control Evolution
- By: Jennifer A. Doudna, Samuel H. Sternberg
- Narrated by: Erin Bennett
- Length: 9 hrs and 22 mins
- Unabridged
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Not since the atomic bomb has a technology so alarmed its inventors that they warned the world about its use. Not, that is, until the spring of 2015, when biologist Jennifer Doudna called for a worldwide moratorium on the use of the new gene-editing tool CRISPR - a revolutionary new technology that she helped create - to make heritable changes in human embryos.
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In to the abyss we ascend, a scary future
- By Philomath on 06-17-17
By: Jennifer A. Doudna, and others
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Where Does It Hurt?
- An Entrepreneur's Guide to Fixing Health Care
- By: Jonathan Bush, Stephen Baker
- Narrated by: Patrick Lawlor
- Length: 7 hrs and 21 mins
- Unabridged
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A bold new remedy for the sprawling and wasteful health care industry. In this provocative book, Jonathan Bush, cofounder and CEO of athenahealth, calls for a revolution in health care to give customers more choices, freedom, power, and information, and at far lower prices.
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No critical thinking
- By Steve from MD on 07-31-14
By: Jonathan Bush, and others
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Epic Measures
- One Doctor. Seven Billion Patients.
- By: Jeremy N. Smith
- Narrated by: Patrick Lawlor
- Length: 10 hrs and 12 mins
- Unabridged
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Moneyball meets medicine in this remarkable chronicle of one of the greatest scientific quests of our time - the groundbreaking program to answer the most essential question for humanity: How do we live and die? - and the visionary mastermind behind it.
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Fabulously insightful read!
- By Dr. Jack E. Fincham on 10-08-15
By: Jeremy N. Smith
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The Problem of Alzheimer's
- How Science, Culture, and Politics Turned a Rare Disease into a Crisis and What We Can Do About It
- By: Jason Karlawish
- Narrated by: Jason Karlawish, Jonathan Todd Ross
- Length: 13 hrs and 41 mins
- Unabridged
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In 2020, an estimated 5.8 million Americans had Alzheimer’s, and more than half a million died because of the disease and its devastating complications. Sixteen million caregivers are responsible for paying as much as half of the $226 billion annual costs of their care. As more people live beyond their 70s and 80s, the number of patients will rise to an estimated 13.8 million by 2025. Part case studies, part meditation on the past, present and future of the disease, The Problem of Alzheimer's traces Alzheimer’s from its beginnings to its recognition as a crisis.
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A must read
- By kara kuntz on 05-20-21
By: Jason Karlawish
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Africa Rise and Shine
- By: Jim Ovia
- Narrated by: David Applefield
- Length: 4 hrs and 8 mins
- Unabridged
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The road to success is rarely linear and never easy. But with courage, hard work, perseverance, and dedication to duty, Jim Ovia, founder and chairman of Zenith Bank, proves we can achieve the unthinkable. Jim has been called the Godfather of Banking by Forbes Africa. And this should be no surprise. In a time of tension between military and civilian regimes, periods of incredible economic instability, and a decaying infrastructure, Jim founded Zenith Bank in Nigeria.
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Very inspiring
- By Henry on 06-10-23
By: Jim Ovia
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Out-Innovate
- How Global Entrepreneurs - from Delhi to Detroit - Are Rewriting the Rules of Silicon Valley
- By: Alexandre Lazarow
- Narrated by: Dennis Boutsikaris, Alexandre Lazarow
- Length: 7 hrs and 42 mins
- Unabridged
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As venture capitalist Alexandre Lazarow shows in this insightful and instructive book, this Silicon Valley "gospel" is due for a refresh - and it comes from what he calls the "frontier", the growing constellation of startup ecosystems, outside of the Valley and other major economic centers, that now stretches across the globe. The frontier is a truly different world where startups often must cope with political or economic instability and lack of infrastructure, and where there might be little or no access to angel investors, venture capitalists, or experienced employee pools.
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Great material for SF and Frontier entrepreneurs!
- By Brett Fulmer on 04-15-20
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Happy Accidents
- Serendipity in Major Medical Breakthroughs in the Twentieth Century
- By: Morton A. Meyers
- Narrated by: Richard Waterhouse
- Length: 12 hrs and 37 mins
- Unabridged
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Happy Accidents is a fascinating, entertaining, and highly accessible look at the surprising role serendipity has played in some of the most important medical discoveries in the 20th century. What do penicillin, chemotherapy drugs, X-rays, Valium, the Pap smear, and Viagra have in common? They were each discovered accidentally, stumbled upon in the search for something else. In discussing medical breakthroughs, Dr. Morton Meyers makes a cogent, highly engaging argument for a more creative, rather than purely linear, approach to science. And it may just save our lives!
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Don't waste your money!
- By Amazon Customer on 03-20-16
By: Morton A. Meyers
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Sicker, Fatter, Poorer
- The Urgent Threat of Hormone-Disrupting Chemicals on Our Health and Future . . . and What We Can Do About It
- By: Leonardo Trasande MD MPP
- Narrated by: Leonardo Trasande MD MPP
- Length: 6 hrs and 44 mins
- Unabridged
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Lurking in our homes, hiding in our offices, and polluting the air we breathe is something sinister. Something we’ve turned a blind eye to for far too long. Dr. Leonardo Trasande, a pediatrician, professor, and world-renowned researcher, tells the story of how our everyday surroundings are making us sicker, fatter, and poorer. Through a blend of narrative, scientific detective work, and concrete information about the connections between chemicals and disease, he reveals what we can do to protect ourselves and our families in the short-term, and how we can help bring the change we deserve.
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The Must Read Book of 2019 is here early on Audio!
- By Ryan S on 12-21-18
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Rigor Mortis
- How Sloppy Science Creates Worthless Cures, Crushes Hope, and Wastes Billions
- By: Richard Harris
- Narrated by: Joe Delafield
- Length: 5 hrs and 56 mins
- Unabridged
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American taxpayers spend $30 billion annually funding biomedical research, but over half of these studies can't be replicated due to poor experimental design, improper methods, and sloppy statistics. Bad science doesn't just hold back medical progress, it can sign the equivalent of a death sentence for terminal patients. In Rigor Mortis, Richard Harris explores these urgent issues with vivid anecdotes, personal stories, and interviews with the top biomedical researchers. We need to fix our dysfunctional biomedical system - before it's too late.
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Eye opening introduction to biomedical R&D
- By Amazon Customer on 09-18-18
By: Richard Harris
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The Prosperity Paradox
- How Innovation Can Lift Nations out of Poverty
- By: Clayton M. Christensen, Efosa Ojomo, Karen Dillon
- Narrated by: Mike Chamberlain
- Length: 9 hrs and 37 mins
- Unabridged
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Clayton M. Christensen, the author of such business classics as The Innovator’s Dilemma and the New York Times best-seller How Will You Measure Your Life, and coauthors Efosa Ojomo and Karen Dillon reveal why so many investments in economic development fail to generate sustainable prosperity and offers a groundbreaking solution for true and lasting change.
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Simplistic, lack of insights
- By D. Cameron on 05-24-21
By: Clayton M. Christensen, and others
What listeners say about Inside the Orphan Drug Revolution
Average customer ratingsReviews - Please select the tabs below to change the source of reviews.
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- Fox2
- 08-05-24
Excellent learning onorphan drugs and beyond
highly recommended to learn about orphan drugs and pharmaceutical industry, R&D, cost and beyond to appreciate all aspects of science and business
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- Colin G.
- 06-01-23
powerful and insightful
a captivating history of entrepreneurship, scientific exploration, and patient centric health breakthroughs. A great read!
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- ItsBeenReal
- 05-29-24
Great information positively presented on a hard subject
Narration did not add or detract from the story. maybe because I am an insider, but I loved the material and found it very informative
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