Download or read book Potential Therapeutic Strategies for Muscular Dystrophy written by Gisela Gaina and published by BoD – Books on Demand. This book was released on 2023-11-02 with total page 116 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Muscular Dystrophy written by Gisela Gaina and published by BoD – Books on Demand. This book was released on 2020-12-23 with total page 117 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book The Muscular Dystrophies written by Alan E. H. Emery and published by . This book was released on 2001 with total page 338 pages. Available in PDF, EPUB and Kindle. Book excerpt: The muscular dystrophies are an important group of inherited disorders. They are characterized by muscle wasting and weakness, but vary considerably in their clinical manifestations and severity. This text reviews our understanding of the most important of these disorders. In many instances, the genes and protein products responsible for the dystrophies have been identified and it is now possible to establish a precise diagnosis, detect preclinical cases, identify carriers and offer prenatal dianostic testing. The book goes on to describe the opportunities for management of the symptoms through respiratory care, physiotherapy and surgical correction of contracture, and examines the potential, in the future, for effective treatment utilizing the new techniques of gene and cell therapy. Professor Emery has invited chapters from the leading international experts in the field providing a unique insight into the current situation and the hopes for the future.
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.
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
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.
Download or read book Muscular Dystrophy written by Madhuri Hegde and published by BoD – Books on Demand. This book was released on 2012-05-09 with total page 558 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Myoblast Transfer Therapy written by Robert C. Griggs and published by Springer. This book was released on 2012-03-28 with total page 330 pages. Available in PDF, EPUB and Kindle. Book excerpt: I am pleased to introduce this volume on Myoblast Transfer Therapy on behalf of the Muscular Dystrophy Association and all of its Advisory Committees. The international conference which led to this volume brought together leading basic scientists and clinical investigators for the purpose of coordinating the development of this new field in the fight against muscular dystrophy. The Muscular Dystrophy Association is the nation's most rapidly growing voluntary health agency in terms of its programs of patient care, research, and professional and public education. Success is attributable to its National Chairman, Jerry Lewis, to its effective corporate membership, and to the many physicians and scientists who give their time freely to advise on policies, to review grant applications, and to participate in meetings such as this. I should like to acknowledge a large number of other individuals to whom we are indebted: the broad segment of the American public which continually and generously supports our spectrum of services. The Muscular Dystrophy Association, next year, should raise in excess of $115,000,000. These contributions are derived from more than 10 million American families. These families are not only pledging their money but expressing their hopes that we will find answers to the tragic problem of neuromuscular disease. We are confident that the fruits of this meeting will move the frontier of research forward toward that goal.
Download or read book Neuromuscular Disorders of Infancy Childhood and Adolescence written by H. Royden Jones and published by Butterworth-Heinemann. This book was released on 2003 with total page 1384 pages. Available in PDF, EPUB and Kindle. Book excerpt: Written by a panel of world authorities, this comprehensive text is the only book of its kind, covering the full range of neuromuscular diseases seen in children. It explains how childhood neuromuscular diseases differ from those in adult patients, and it provides clinicians with all the knowledge they need to successfully diagnose and treat their pediatric patients.
Download or read book Selected Health Conditions and Likelihood of Improvement with Treatment written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-07-12 with total page 319 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Social Security Administration (SSA) administers two programs that provide disability benefits: the Social Security Disability Insurance (SSDI) program and the Supplemental Security Income (SSI) program. SSDI provides disability benefits to people (under the full retirement age) who are no longer able to work because of a disabling medical condition. SSI provides income assistance for disabled, blind, and aged people who have limited income and resources regardless of their prior participation in the labor force. Both programs share a common disability determination process administered by SSA and state agencies as well as a common definition of disability for adults: "the inability to engage in any substantial gainful activity by reason of any medically determinable physical or mental impairment which can be expected to result in death or which has lasted or can be expected to last for a continuous period of not less than 12 months." Disabled workers might receive either SSDI benefits or SSI payments, or both, depending on their recent work history and current income and assets. Disabled workers might also receive benefits from other public programs such as workers' compensation, which insures against work-related illness or injuries occurring on the job, but those other programs have their own definitions and eligibility criteria. Selected Health Conditions and Likelihood of Improvement with Treatment identifies and defines the professionally accepted, standard measurements of outcomes improvement for medical conditions. This report also identifies specific, long-lasting medical conditions for adults in the categories of mental health disorders, cancers, and musculoskeletal disorders. Specifically, these conditions are disabling for a length of time, but typically don't result in permanently disabling limitations; are responsive to treatment; and after a specific length of time of treatment, improve to the point at which the conditions are no longer disabling.
Download or read book Peptides for Youth written by Susan Valle and published by Springer Science & Business Media. This book was released on 2009-06-23 with total page 703 pages. Available in PDF, EPUB and Kindle. Book excerpt: The American Peptide Society (APS) provides a forum for advancing and promoting knowledge of the chemistry and biology of peptides. The approximately one thousand members of the Society come from North America and from more than thirty other countries throughout the world. Establishment of the APS was a result of the rapid worldwide growth that has occurred in peptide-related research, and of the increasing interaction of peptide scientists with virtually all fields of science. Peptides for Youth: The Proceedings of the the 20th American Peptide Symposium will highlight many of the recent developments in peptide science, with a particular emphasis on how these advances are being applied to basic problems in biology and medicine. The 20th American Peptide Symposium will take place June 26 - 30, 2007 in Montreal, Canada.
Download or read book Disorders of Voluntary Muscle written by George Karpati and published by Cambridge University Press. This book was released on 2001-07-12 with total page 800 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rewritten and redesigned, this remains the one essential text on the diseases of skeletal muscle.
Download or read book Muscular Dystrophy written by Madhuri Hegde and published by IntechOpen. This book was released on 2012-05-09 with total page 556 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
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.
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.
Download or read book Muscle Regeneration written by Alexander Mauro and published by Raven Press (ID). This book was released on 1979 with total page 582 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Muscle Homeostasis and Regeneration written by Antonio Musarò and published by MDPI. This book was released on 2020-11-20 with total page 500 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book is a collection of original research and review articles addressing the intriguing field of the cellular and molecular players involved in muscle homeostasis and regeneration. One of the most ambitious aspirations of modern medical science is the possibility of regenerating any damaged part of the body, including skeletal muscle. This desire has prompted clinicians and researchers to search for innovative technologies aimed at replacing organs and tissues that are compromised. In this context, the papers, collected in this book, addressing a specific aspects of muscle homeostasis and regeneration under physiopathologic conditions, will help us to better understand the underlying mechanisms of muscle healing and will help to design more appropriate therapeutic approaches to improve muscle regeneration and to counteract muscle diseases.
