Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development.
This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development.
This title is ideal for graduate students/postdocs and principal investigators who are already in the SMA field and need to keep updated on recent findings and approaches, and for those who are new to, or would like to join, the field. Likewise, users will find an excellent source of reading for biotech/pharma scientists, clinical researchers, and practitioners, regulators, and patients and their advocacy organizations. Furthermore, this book is a handy reference for researchers and clinicians who may want to apply the research strategies and therapeutic approaches in SMA to other rare diseases.
Publisher: Elsevier Science Publishing Co Inc
Number of pages: 506
Weight: 1700 g
Dimensions: 276 x 216 x 30 mm
This comprehensive, state-of-the-art textbook, covering all aspects of SMA, is very timely and should provide a springboard for further efforts and advances in the future. -- Victor Dubowitz, M.D., Ph.D., Emeritus Professor of Paediatrics, University of London, London, UK.
This book is written by experts who contribute to major progress in the fields of SMA from the clinical features, molecular mechanisms, animal models, therapeutic developments to clinical trials. I have no doubt that this book will become an indispensable resource for clinicians and scientists having the goal to cure SMA. -- Judith Melki, M.D., Ph.D., Professor of Medical Genetics, Medicine Faculty, University of Paris 11; Inserm and University of Paris 11, U-788, Kremlin-Bicetre Hospital, Paris, France.
Now there is an important book which chronicles the many paths and diverse approaches taken to understand SMA and develop therapies, a clear strength of the SMA field has been the extensive sharing of data that has advanced the field rapidly. --Arthur H.M. Burghes, Ph.D., Professor, Department of Biological Chemistry and Pharmacology, College of Medicine, The Ohio State University, Columbus, OH, USA.
We have come a long way on the path to therapeutics development for SMA, and safe and effective treatment is now within reach. This book comes at an opportune time to take stock of where we are on this path and to see what the future holds. -- Kenneth H. Fischbeck, M.D., NIH Distinguished Investigator, Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.