Orphan Diseases And Orphan Drugs
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Orphan Diseases And Orphan Drugs
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Author : I. Herbert Scheinberg
language : en
Publisher: Manchester University Press
Release Date : 1986
Orphan Diseases And Orphan Drugs written by I. Herbert Scheinberg and has been published by Manchester University Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 1986 with Medical categories.
Rare Diseases And Orphan Products
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Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2011-04-03
Rare Diseases And Orphan Products written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-04-03 with Medical categories.
Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.
Rare Diseases And Orphan Drugs
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Author : Jules J. Berman
language : en
Publisher: Academic Press
Release Date : 2014-05-26
Rare Diseases And Orphan Drugs written by Jules J. Berman and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-05-26 with Medical categories.
Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the disease process observed in a common disease. This work ties the lessons learned about rare diseases to our understanding of common ones. Chapters covering the number of common diseases are minimized, while rare diseases are introduced as single diseases or as members of diseases classes. After reading this book, readers will appreciate how further research into the rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases, rare or common. Makes rare diseases relevant to clinicians and researchers by tying lessons learned about the rare diseases to our understanding of the common diseases Stresses basic pathologic mechanisms that account for human disease (e.g., disorders of cell development, replication, maintenance, function and structure), that can be understood without prior training in pathology Discusses advanced concepts in molecular biology and genetics in a simple, functional context appropriate for medical trainees and new researchers Offers insights into how further research into rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases
Orphan Drugs And Rare Diseases
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Author : David C Pryde
language : en
Publisher: Royal Society of Chemistry
Release Date : 2014-07-30
Orphan Drugs And Rare Diseases written by David C Pryde and has been published by Royal Society of Chemistry this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-07-30 with Science categories.
Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.
Orphan Drugs
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Author : Elizabeth Hernberg-Ståhl
language : en
Publisher: Elsevier
Release Date : 2013-11-15
Orphan Drugs written by Elizabeth Hernberg-Ståhl and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-11-15 with Medical categories.
This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups.The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey.After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs.The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe
Report Of The National Commission On Orphan Diseases
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Author : National Commission on Orphan Diseases (U.S.)
language : en
Publisher:
Release Date : 1989
Report Of The National Commission On Orphan Diseases written by National Commission on Orphan Diseases (U.S.) and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1989 with Federal aid to medical research categories.
Orphan Drugs
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Author : Carolyn H. Asbury
language : en
Publisher: Free Press
Release Date : 1985
Orphan Drugs written by Carolyn H. Asbury and has been published by Free Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 1985 with Drugs categories.
Orphan Drugs And Rare Diseases
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Author : David C Pryde
language : en
Publisher: Royal Society of Chemistry
Release Date : 2014
Orphan Drugs And Rare Diseases written by David C Pryde and has been published by Royal Society of Chemistry this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014 with Medical categories.
This book provides an up-to-date monograph on the drug discovery and regulatory elements of therapuetics used to treat rare or "orphan" diseases.
Introduction To Basics Of Pharmacology And Toxicology
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Author : Gerard Marshall Raj
language : en
Publisher: Springer Nature
Release Date : 2019-11-16
Introduction To Basics Of Pharmacology And Toxicology written by Gerard Marshall Raj and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-11-16 with Medical categories.
This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.
Preliminary Report Of The Survey On Drugs For Rare Disease
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Author :
language : en
Publisher:
Release Date : 1982
Preliminary Report Of The Survey On Drugs For Rare Disease written by and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1982 with Drugs categories.