Geraghty james a (53 résultats)

Hardback. Etat : Very Good. 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.…

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Hardback. Etat : Very Good. 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.…

Etat : very_good.

Etat : good. All pages and cover are intact including the dust cover, if applicable . Spine may show signs of wear. Pages may include limited notes and highlighting. May include "From the library of" labels. Shrink wrap, dust covers, or boxed set case may be missing. Item may be missing bundled media.…

Hardcover. Etat : Very Good. No Jacket. May have limited writing in cover pages. Pages are unmarked. ~ ThriftBooks: Read More, Spend Less.

Etat : new.

hardcover. Etat : Very Good. Connecting readers with great books since 1972! Used books may not include companion materials, and may have some shelf wear or limited writing. We ship orders daily and Customer Service is our top priority.

paperback. Etat : Fine. Sealed in original plastic but plastic is ripped.

hardcover. Etat : As New. Ships Out Tomorrow.

hardcover. Etat : New. New, unread, and unused.

paperback. Etat : New.

Etat : As New. Unread book in perfect condition.

Hardcover. Etat : Very Good. Hardcover (no dust jacket; printed boards). Very good condition. From the offices of a world-leading nuclear medicine research establishment. With 43 figures, 18 in colour. Spine ends and boards' corners are bumped and scuffed, and a couple of other minor scuffs to spine. Pages are sound and clear. TA. Used.…

Etat : New.

HRD. Etat : New. New Book. Shipped from UK. Established seller since 2000.

Hardback. Etat : New.

Hardcover. Etat : New. Brand new book, sourced directly from publisher. Dispatch time is 24-48 hours from our warehouse. Book will be sent in robust, secure packaging to ensure it reaches you securely.

HRD. Etat : New. New Book. Shipped from UK. Established seller since 2000.

Paperback. Etat : New.

Etat : very good. Gut/Very good: Buch bzw. Schutzumschlag mit wenigen Gebrauchsspuren an Einband, Schutzumschlag oder Seiten. / Describes a book or dust jacket that does show some signs of wear on either the binding, dust jacket or pages.

Hardcover. Etat : Brand New. 264 pages. 9.37x6.38x0.94 inches. In Stock.

Hardback. Etat : New.

Etat : New.

Etat : New. 2022. Hardcover. . . . . .

Etat : New. In English.

Etat : As New. Unread book in perfect condition.

Etat : New.

Etat : New. 2022. Hardcover. . . . . . Books ship from the US and Ireland.

Paperback. Etat : Brand New. 1st edition. 265 pages. 9.00x6.00x0.75 inches. In Stock.