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Muscular Dystrophy - Research Update and Therapeutic Strategies is for students, researchers, and clinicians interested in muscular dystrophies who want to improve their knowledge of these complex genetic diseases. The book includes information about the genetics of various types of muscular dystrophies as well as explores new and current therapeutic strategies that aim to ameliorate symptoms and improve patients' quality of life and life expectancy. In addition, this book reviews information on current clinical trials for muscular dystrophies and presents a framework for what to consider during the design of these trials.
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The book provides a comprehensive overview of the different forms of muscular dystrophy (MD), including potential therapeutic interventions and the advantages associated with repurposing pharmaceuticals for treating these conditions. In addition, the book examines the effects of physical training on improving symptoms as well as patient quality of life and life span. The target audience of this book comprises students, researchers, and doctors with an interest in the field of muscular dystrophy.
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Muscular dystrophy --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases
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Muscular dystrophy. --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases
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The field of muscular dystrophies has expanded significantly with the discovery of the genetic defects and protein products underlying these disorders. New treatments such as antisense oligonucleotides and siRNAs and gene therapies are now in clinical application and in trials. As clinical trials increase, outcome measurement becomes very important, and more validated tools are being developed. This book provides a comprehensive review of these disorders. Clinical features, diagnostic testing including genetic testing of these disorders, the basis and utility of genetic testing, and the basis of genetic therapies are all covered in detail. This book will provide neuromuscular neurologists, general neurologists, and neuromuscular fellows with a much needed update in the field.
Neurology. --- Diagnosis. --- Therapeutics. --- Nervous system --- Neurological Disorders. --- Diseases. --- Muscular dystrophy.
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Skeletal muscle is a highly plastic organ that is modulated by various pathways controlling protein turnover. Muscle loss is common in muscular dystrophy, in which marked loss of various proteins such as the dystrophin-glycoprotein complex occurs around muscle fibers. This book provides a comprehensive overview of the various muscular dystrophies, including characteristics, diagnosis, and classification. General treatment of drugs (e.g. corticosteroids) and physical therapy for muscular dystrophies are discussed. In addition, current applications for cell and tissue engineering using muscle stem cells or gene therapy are introduced. This book also deals with the recent advances in appropriate models of drug screening using cell cultures or mammalian organs in vitro in this field.
Muscular dystrophy. --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases --- Medicine --- Medical Genetics --- Molecular Genetics --- Health Sciences
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With more than 30 different types and subtypes known and many more yet to be classified and characterized, muscular dystrophy is a highly heterogeneous group of inherited neuromuscular disorders. This book provides a comprehensive overview of the various types of muscular dystrophies, genes associated with each subtype, disease diagnosis, management as well as available treatment options. Though each different type and subtype of muscular dystrophy is associated with a different causative gene, the majority of them have overlapping clinical presentations, making molecular diagnosis inevitable for both disease diagnosis as well as patient management. This book discusses the currently available diagnostic approaches that have revolutionized clinical research. Pathophysiology of the different muscular dystrophies, multifaceted functions of the involved genes as well as efforts towards diagnosis and effective patient management, are also discussed. Adding value to the book are the included reports on ongoing studies that show a promise for future therapeutic strategies.
Muscular dystrophy. --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases --- Medical genetics
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Providing specific guidance for people with muscular dystrophy and their families, this accessible work answers many questions asked about how and why it occurs, and its affect on the life of a recently diagnosed child.
Muscular dystrophy --- Internal medicine. --- Medicine, Internal --- Medicine --- Muscular dystrophies --- Dystrophy --- Genetic disorders --- Neuromuscular diseases
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This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact
Gait --- Cerebral Palsy --- pediatric neurology --- MRI --- Upper limb --- Ataxia --- tDCS --- Duchenne muscular dystrophy
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