This book presents recent advances in translational research on muscular dystrophy (MD) to physicians and researchers, including cutting-edge research on the disease such as regenerative medicine, next-generation DNA sequencing, and nucleic acid therapies. It also describes the current systems for clinical trials and MD patient databases, resources, which will support the early realization of clinical application and improve patients’ quality of life. MD is the one of the most widely known inherited neuromuscular diseases and is classified into diverse types by symptoms, age of onset, mode of inheritance, and clinical progression. With the development of molecular biology, the occurrence mechanisms of each type of MD are gradually being elucidated. Although there is no known permanent cure yet, the stage of treatment research has now advanced to clinical trials.

In this new book, noted geneticist and veteran OUP author, Moyra Smith, present a comprehensive critical review of the translation of genetic and genomic research into health care. Dr. Smith's motivation for writing is driven by the gap that exists between the rather amazing discoveries in medical genetics and genomics at basic science levels and the translation to disease management in single gene disorders, specific genetic syndromes, and complex genetic diseases. She also examines information technology in genetic medicine, sociocultural factors that impact provision of medical care, and medical education issues with regard to translational genetics in order to help prepare a work force that is better able to utilize evidence-based medicine and to accomodate the rapid changes in genetic and genomic health care.

This book presents recent advances in translational research on muscular dystrophy (MD) to physicians and researchers, including cutting-edge research on the disease such as regenerative medicine, next-generation DNA sequencing, and nucleic acid therapies. It also describes the current systems for clinical trials and MD patient databases, resources, which will support the early realization of clinical application and improve patients' quality of life. MD is the one of the most widely known inherited neuromuscular diseases and is classified into diverse types by symptoms, age of onset, mode of inheritance, and clinical progression. With the development of molecular biology, the occurrence mechanisms of each type of MD are gradually being elucidated. Although there is no known permanent cure yet, the stage of treatment research has now advanced to clinical trials.

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.

Many voluntary health organizations fund translational research. An increasing number of these organizations are looking at venture philanthropy as a critical way to advance their missions of helping patients and working to cure disease. A wide range of participants gathered on October 3, 2008 at the Beckman Center of the National Academies of Science for a workshop titled "Venture Philanthropy Strategies Used by Patient Organizations to Support Translational Research." Participants with experience in venture philanthropy shared their experiences and lessons learned in order to improve efficiency and effectiveness in translational research.

There are 8 different known types of this disorder, and some Muscular Dystrophy can actually be discovered during pregnancy, according to HRF. This guidebook provides essential information on MD, but also serves as a historical survey, by providing information on the controversies surrounding its causes, and first-person narratives by people coping with MD. Patients, family members, or caregivers explain the condition from their own experience. The symptoms, causes, and treatments are explained in detail. Essential to anyone trying to learn about diseases and conditions, the alternative treatments are explored. Each essay is carefully edited and presented with an introduction, so that they are accessible for student researchers and readers.

Translational Regenerative Medicine is a reference book that outlines the life cycle for effective implementation of discoveries in the dynamic field of regenerative medicine. By addressing science, technology, development, regulatory, manufacturing, intellectual property, investment, financial, and clinical aspects of the field, this work takes a holistic look at the translation of science and disseminates knowledge for practical use of regenerative medicine tools, therapeutics, and diagnostics. Incorporating contributions from leaders in the fields of translational science across academia, industry, and government, this book establishes a more fluid transition for rapid translation of research to enhance human health and well-being. - Provides formulaic coverage of the landscape, process development, manufacturing, challenges, evaluation, and regulatory aspects of the most promising regenerative medicine clinical applications - Covers clinical aspects of regenerative medicine related to skin, cartilage, tendons, ligaments, joints, bone, fat, muscle, vascular system, hematopoietic /immune system, peripheral nerve, central nervous system, endocrine system, ophthalmic system, auditory system, oral system, respiratory system, cardiac system, renal system, hepatic system, gastrointestinal system, genitourinary system - Identifies effective, proven tools and metrics to identify and pursue clinical and commercial regenerative medicine