Muscle Gene Therapy

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Publisher : Springer Science & Business Media
ISBN 13 : 144191207X
Total Pages : 281 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Muscle Gene Therapy by : Dongsheng Duan

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.

NORD Guide to Rare Disorders

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Publisher : Lippincott Williams & Wilkins
ISBN 13 : 9780781730631
Total Pages : 982 pages
Book Rating : 4.7/5 (36 download)

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Book Synopsis NORD Guide to Rare Disorders by : National Organization for Rare Disorders

Download or read book NORD Guide to Rare Disorders written by National Organization for Rare Disorders and published by Lippincott Williams & Wilkins. This book was released on 2003 with total page 982 pages. Available in PDF, EPUB and Kindle. Book excerpt: NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words offers physicians valuable assistance in finding the information they need quickly.

Neuromuscular Disorders of Infancy, Childhood, and Adolescence

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Author :
Publisher : Elsevier
ISBN 13 : 0124171273
Total Pages : 1156 pages
Book Rating : 4.1/5 (241 download)

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Book Synopsis Neuromuscular Disorders of Infancy, Childhood, and Adolescence by : Basil T. Darras

Download or read book Neuromuscular Disorders of Infancy, Childhood, and Adolescence written by Basil T. Darras and published by Elsevier. This book was released on 2014-12-03 with total page 1156 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular disorders are diagnosed across the lifespan and create many challenges especially with infants, children and adolescents. This new edition of the definitive reference, edited by the established world renowned authorities on the science, diagnosis and treatment of neuromuscular disorders in childhood is a timely and needed resource for all clinicians and researchers studying neuromuscular disorders, especially in childhood. The Second Edition is completely revised to remain current with advances in the field and to insure this remains the standard reference for clinical neurologists and clinical research neurologists. The Second Edition retains comprehensive coverage while shortening the total chapter count to be an even more manageable and effective reference. - Carefully revised new edition of the classic reference on neuromuscular disorders in infancy, childhood and adolescence. - Definitive coverage of the basic science of neuromuscular disease and the latest diagnosis and treatment best practices. - Includes coverage of clinical phenomenology, electrophysiology, histopathology, molecular genetics and protein chemistry

Muscular Dystrophy

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Author :
Publisher : Springer
ISBN 13 : 3319173626
Total Pages : 206 pages
Book Rating : 4.3/5 (191 download)

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Book Synopsis Muscular Dystrophy by : Raymond A. Huml

Download or read book Muscular Dystrophy written by Raymond A. Huml and published by Springer. This book was released on 2015-07-16 with total page 206 pages. Available in PDF, EPUB and Kindle. Book excerpt: This practical and concise guide offers an overview of muscular dystrophy's complicated features, treatment options and general resources. New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long term patient care. Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances. Muscular Dystrophy offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient.

Duchenne Muscular Dystrophy

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Author :
Publisher : OUP Oxford
ISBN 13 : 0191503665
Total Pages : 300 pages
Book Rating : 4.1/5 (915 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Alan E. H. Emery

Download or read book Duchenne Muscular Dystrophy written by Alan E. H. Emery and published by OUP Oxford. This book was released on 2015-02-19 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy.

Genome Editing in Neurosciences

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Publisher :
ISBN 13 : 9781013268564
Total Pages : 128 pages
Book Rating : 4.2/5 (685 download)

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Book Synopsis Genome Editing in Neurosciences by : Rudolf Jaenisch

Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by . This book was released on 2020-10-08 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.

Rare Disease Drug Development

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Publisher : Springer Nature
ISBN 13 : 3030786056
Total Pages : 418 pages
Book Rating : 4.0/5 (37 download)

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Book Synopsis Rare Disease Drug Development by : Raymond A. Huml

Download or read book Rare Disease Drug Development written by Raymond A. Huml and published by Springer Nature. This book was released on 2021-11-08 with total page 418 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.

Duchenne Muscular Dystrophy

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Publisher : Humana Press
ISBN 13 : 9781493973736
Total Pages : 287 pages
Book Rating : 4.9/5 (737 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Camilla Bernardini

Download or read book Duchenne Muscular Dystrophy written by Camilla Bernardini and published by Humana Press. This book was released on 2017-10-25 with total page 287 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on “Omics” techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder.

Ferri's Clinical Advisor 2020 E-Book

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Publisher : Elsevier Health Sciences
ISBN 13 : 0323679773
Total Pages : 7615 pages
Book Rating : 4.3/5 (236 download)

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Book Synopsis Ferri's Clinical Advisor 2020 E-Book by : Fred F. Ferri

Download or read book Ferri's Clinical Advisor 2020 E-Book written by Fred F. Ferri and published by Elsevier Health Sciences. This book was released on 2019-06-01 with total page 7615 pages. Available in PDF, EPUB and Kindle. Book excerpt: Significantly updated with the latest developments in diagnosis and treatment recommendations, Ferri's Clinical Advisor 2020 features the popular "5 books in 1" format to organize vast amounts of information in a clinically relevant, user-friendly manner. This efficient, intuitive format provides quick access to answers on 1,000 common medical conditions, including diseases and disorders, differential diagnoses, and laboratory tests – all reviewed by experts in key clinical fields. Updated algorithms, along with hundreds of new figures, tables, and boxes, ensure that you stay current with today's medical practice. - Contains significant updates throughout, covering all aspects of current diagnosis and treatment. - Features 27 all-new topics including chronic traumatic encephalopathy, medical marijuana, acute respiratory failure, gallbladder carcinoma, shift work disorder, radial tunnel syndrome, fertility preservation in women, fallopian tube cancer, primary chest wall cancer, large-bowel obstruction, inguinal hernia, and bundle branch block, among others. - Includes a new appendix covering Physician Quality Reporting System (PQRS) Measures. - Provides current ICD-10 insurance billing codes to help expedite insurance reimbursements. - Patient Teaching Guides for many of the diseases and disorders are included, most available in both English and Spanish versions, which can be downloaded and printed for patients.

Muscular Dystrophy

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Author :
Publisher :
ISBN 13 :
Total Pages : 26 pages
Book Rating : 4.:/5 (319 download)

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Book Synopsis Muscular Dystrophy by : National Institute of Neurological Diseases and Stroke

Download or read book Muscular Dystrophy written by National Institute of Neurological Diseases and Stroke and published by . This book was released on 1971 with total page 26 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Duchenne Muscular Dystrophy

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Author :
Publisher : CRC Press
ISBN 13 : 0849374456
Total Pages : 491 pages
Book Rating : 4.8/5 (493 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Jeffrey S. Chamberlain

Download or read book Duchenne Muscular Dystrophy written by Jeffrey S. Chamberlain and published by CRC Press. This book was released on 2006-02-27 with total page 491 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This authoritative guide provides a clear overview of the latest current and experimental approaches to the treatment of DMD and examines the clinical, genetic, and pathophysiological aspects of the disease i

Duchenne Muscular Dystrophy

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Publisher :
ISBN 13 : 9781634821537
Total Pages : 200 pages
Book Rating : 4.8/5 (215 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Samuel Alexandre Almeida Honorio

Download or read book Duchenne Muscular Dystrophy written by Samuel Alexandre Almeida Honorio and published by . This book was released on 2015-04-01 with total page 200 pages. Available in PDF, EPUB and Kindle. Book excerpt: A child with Duchenne Muscular Dystrophy (DMD) faces a childhood and adolescence with a disability that develops gradually. This book intends to expose the problems of children with DMD. The authors discuss situations related to the disease in an attempt to provide some quality and improvement of life for those affected since DMD is a degenerative disease which affects muscle. This book offers a historical study of muscular dystrophy. It also shows some profiles of excellent clinicians and scientists that have contributed to this book with a description of muscular dystrophy from the clinical picture, to the latest techniques in genetics. Also included in the chapters are resolutions of several approaches to DMD, like scales, in order to anticipate how to manage the disease. This book relates some techniques of moderate exercise, like Yoga and water exercises, that are compliable with some advantages in mobility. The book concludes with some current trends and what can be done in the future. This book can be a good read to everyone that is interested in DMD, and also for the ones who know or provide care for those with this terrible disease.

Muscular Dystrophy

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Author :
Publisher : Nova Science Publishers
ISBN 13 : 9781626184602
Total Pages : 0 pages
Book Rating : 4.1/5 (846 download)

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Book Synopsis Muscular Dystrophy by : Corrado Angelini

Download or read book Muscular Dystrophy written by Corrado Angelini and published by Nova Science Publishers. This book was released on 2013 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscular dystrophies include a heterogeneous series of diseases that range from childhood to adult onset cases, of difficult diagnosis and treatment. This book presents the state of the art in muscular dystrophies, after the molecular revolution.

The Human Genome

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Publisher : Academic Press
ISBN 13 : 0080918654
Total Pages : 600 pages
Book Rating : 4.0/5 (89 download)

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Book Synopsis The Human Genome by : Julia E. Richards

Download or read book The Human Genome written by Julia E. Richards and published by Academic Press. This book was released on 2010-12-12 with total page 600 pages. Available in PDF, EPUB and Kindle. Book excerpt: Significant advances in our knowledge of genetics were made during the twentieth century but in the most recent decades, genetic research has dramatically increased its impact throughout society. Genetic issues are now playing a large role in health and public policy, and new knowledge in this field will continue to have significant implications for individuals and society. Written for the non-majors human genetics course, Human Genetics, Third Edition will increase the genetics knowledge of students who are learning about human genetics for the first time. This thorough revision of the best-selling Human Genome, Second Edition includes entirely new chapters on forensics, stem cell biology, bioinformatics, and societal/ethical issues associated with the field. New special features boxes make connections between human genetics and human health and disease. Carefully crafted pedagogy includes chapter-opening case studies that set the stage for each chapter; concept statements interspersed throughout the chapter that keep first-time students focused on key concepts; and end-of-chapter questions and critical thinking activities. This new edition will contribute to creating a genetically literate student population that understands basic biological research, understands elements of the personal and health implications of genetics, and participates effectively in public policy issues involving genetic information. - Includes topical material on forensics, disease studies, and the human genome project to engage non-specialist students - Full, 4-color illustration program enhances and reinforces key concepts and themes - Uniform organization of chapters includes interest boxes that focus on human health and disease, chapter-opening case studies, and concept statements to engage non-specialist readers

Neuromuscular Imaging

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Publisher : Springer Science & Business Media
ISBN 13 : 1461465524
Total Pages : 432 pages
Book Rating : 4.4/5 (614 download)

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Book Synopsis Neuromuscular Imaging by : Mike P. Wattjes

Download or read book Neuromuscular Imaging written by Mike P. Wattjes and published by Springer Science & Business Media. This book was released on 2013-06-05 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular imaging has increasingly become an important tool in the detection and diagnosis of inherited and acquired neuromuscular disease. This book is a groundbreaking radiological and neurological overview of current methods and applications of imaging—including aspects of neuroimaging and musculoskeletal imaging—in patients with inherited, metabolic, and inflammatory muscle diseases. Imaging features are discussed in the context of clincial presentation, histopathology, therapeutic options and differential diagnosis. World leading expert contributors give a comprehensive and didactic review of neuromuscular disorders and available imaging modalities, each illustrated with numerous figures. Topics discussed include: -Modalities such as ultrasound, CT and MRI -Muscle anatomy and physiology -Clinical applications in hereditary and acquired myopathies -Clinical applications in motor neuron disorders and peripheral nerve imaging

Advances in Eicosanoid Research

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Publisher : Springer Science & Business Media
ISBN 13 : 9783540667476
Total Pages : 300 pages
Book Rating : 4.6/5 (674 download)

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Book Synopsis Advances in Eicosanoid Research by : C.N. Serhan

Download or read book Advances in Eicosanoid Research written by C.N. Serhan and published by Springer Science & Business Media. This book was released on 2000-04-10 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over the last few years, we have witnessed tremendous progress in the field of eicosanoids and their therapeutic applications. Receptor an tagonists for leukotrienes have been tested as anti-inflammatories and are on the market as a treatment for asthma. Receptor agonists for pro stacyclin are being tested for the treatment of peripheral vascular dis ease, and selective inhibitors of cyclooxygenase type II were just ap proved for the treatment of rheumatoid arthritis. All these developments are the culmination of many years and man-hours of careful research. The field has now entered an upswing that will result in novel thera peutic applications within the next 10 years. New molecules and me diators have been identified, new enzymes and pathways elucidated and new therapeutic approaches have emerged. The concept of ei cosanoids as "pro-inflammatory" molecules is being challenged, and their role as regulators is increasingly recognized. In fact, some of these molecules may be important endogenous anti-inflammatory agents.

Research Into the Origin and Treatment of Muscular Dystrophy

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Author :
Publisher :
ISBN 13 :
Total Pages : 184 pages
Book Rating : 4.3/5 (91 download)

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Book Synopsis Research Into the Origin and Treatment of Muscular Dystrophy by : L. P. ten Kate

Download or read book Research Into the Origin and Treatment of Muscular Dystrophy written by L. P. ten Kate and published by . This book was released on 1984 with total page 184 pages. Available in PDF, EPUB and Kindle. Book excerpt: