Rare Diseases and Orphan Products PDF Download
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Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309158060
Category : Medical
Languages : en
Pages : 442
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Book Description
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.
Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309158060
Category : Medical
Languages : en
Pages : 442
Get Book Here
Book Description
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.
Author: Asla Pitkänen
Publisher: Academic Press
ISBN: 012804067X
Category : Medical
Languages : en
Pages : 1180
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Book Description
Models of Seizures and Epilepsy, Second Edition, is a valuable, practical reference for investigators who are searching for the most appropriate laboratory models to address key questions in the field. The book also provides an important background for physicians, fellows, and students, offering insight into the potential for advances in epilepsy research as well as R&D drug development. Contents include the current spectrum of models available to model different epilepsy syndromes, epilepsy in transgenic animals, comorbidities in models of epilepsy, and novel technologies to study seizures and epilepsies in animals. - Provides a comprehensive reference detailing animal models of epilepsy and seizure - Offers insights on the use of novel technologies that can be applied in experimental epilepsy research - Edited by leading experts in the field that provide not only technical reviews of these models but also conceptual critiques - Comments on the strengths and limitations of various models, including their relationship to clinical phenomenology and their value in developing better understanding and treatments
Author: M. Nikanorova
Publisher: John Libbey Eurotext
ISBN: 2742012354
Category :
Languages : en
Pages : 141
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Book Description
All the necessary information on 6 molecules called “orphan drugs” used in the treatment of some epileptic syndromes. More than half of epilepsies start before the age of 20 years and nearly 25% of them are refractory. Two problems arise: - It is essential to treat them so that brain development continues as normally as possible. - There are few drugs available. This work outlines 6 molecules in detail that are specifically used in epileptic encephalopathies. Every aspect of these molecules is discussed, including drug development, indication, efficacy, cost, etc. Drafted by international experts in the field of epileptology, this book provides all the necessary information on orphan drugs and their clinical use.
Author: Vibhangini S. Wasade
Publisher: Cambridge University Press
ISBN: 1108718906
Category : Medical
Languages : en
Pages : 501
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Book Description
A comprehensive, accessible synthesis of current information on epilepsy for medical trainees and physicians preparing for board certification.
Author: Dieter Schmidt
Publisher: Cambridge University Press
ISBN: 1316409716
Category : Medical
Languages : en
Pages : 263
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Book Description
Epilepsy is amongst the most frequently encountered of neurological disorders, yet its diagnosis and management is fraught with pitfalls. Issues surrounding differential diagnosis, seizure type, underlying causes, EEG interpretations, treatment options and prognoses can often trip-up clinicians. Common Epilepsy Pitfalls: Case-Based Learning, is a comprehensive guide to anticipating and managing the pitfalls in the diagnosis and management of epilepsy. Real-life cases are presented along with pragmatic recommendations for courses of action that guide the reader through accurate and effective epilepsy diagnosis and treatment. Cases have been chosen to answer key questions such as: the first seizure – is it epilepsy? Which drug? When should stopping treatment be considered? What to do in an epileptic emergency? Containing detailed coverage of psychiatric, social and family issues – and authored by world experts in epilepsy management – this text is invaluable for neurologists, internists, family practitioners and advanced medical students.
Author: Michael Duchowny
Publisher: McGraw Hill Professional
ISBN: 0071641327
Category : Medical
Languages : en
Pages : 526
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Book Description
Market: Neurologists and pediatricians Diagnostic and treatment algorithms appear throughout Includes sections on comorbidities and monotherapy vs. polytherapy
Author: Gonzalo Alarcón
Publisher: Cambridge University Press
ISBN: 0521691583
Category : Medical
Languages : en
Pages : 641
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Book Description
Covers all aspects of epilepsy, from basic mechanisms to diagnosis and management, as well as legal and social considerations.
Author: Brett E. Skolnick
Publisher: Academic Press
ISBN: 0128041013
Category : Medical
Languages : en
Pages : 408
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Book Description
Handbook of Neuroemergency Clinical Trials, Second Edition, focuses on the practice of clinical trials in acute neuroscience populations, or what have been called neuroemergencies. Neuroemergencies are complex, life-threatening diseases and disorders, often with devastating consequences, including death or disability. The overall costs are staggering in terms of annual incidence and costs associated with treatment and survival, yet despite their significance as public health issues, there are few drugs and devices available for definitive treatment. The book focuses on novel therapies and the unique challenges their intended targets pose for the design and analysis of clinical trials. This volume provides neurologists, neuroscientists, and drug developers with a more complete understanding of the scientific and medical issues of relevance in designing and initiating clinical development plans for novel drugs intended for acute neuroscience populations. The editors provide the best understanding of the pitfalls associated with acute CNS drug development and the best information on how to approach and solve issues that have plagued drug development. - Presents a comprehensive overview on clinical trials and drug development challenges in acute neuroscience populations - Provides neurologists, neuroscientists and drug developers with a complete understanding of scientific and medical issues related to designing clinical trials - Edited by leaders in the field who have designed and managed over 50 neuroemergency clinical trials
Author: Farid A. Badria
Publisher: BoD – Books on Demand
ISBN: 1839685204
Category : Medical
Languages : en
Pages : 236
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Book Description
Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry.
Author: David Pryde
Publisher: Royal Society of Chemistry
ISBN: 1782624201
Category : Science
Languages : en
Pages : 487
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Book Description
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.
