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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...
This book provides an accessible guide to neuromuscular disorders using case scenarios from the world-renowned MRC Centre for Neuromuscular Diseases at the National Hospital, Queen Square, London, UK. Fifty genetic and acquired disorders are presented in a practical, easy-to-read format, including those that are common and also some which are rare. Each case covers the history, examination and investigations, including neurophysiology, neuroradiology and neuropathology if appropriate. Discussions of each case include the differential diagnosis, useful clinical pointers and a brief summary of the management of the condition. Neuromuscular Disease: Case Studies from Queen Square is aimed at neurology trainees and consultant general neurologists.
This comprehensive encyclopedia supplies the reader with concise information on the molecular pathophysiology of disease. Entries include defined diseases (such as Parkinson's disease) as well as pathophysiological entities (such as tremor). The 1,200 essays are brilliantly structured to allow rapid retrieval of the desired information. For more detailed reading, each entry is followed by up to five references. Individual entries are written by leading experts in the respective area of research to ensure state-of-the-art descriptions of the mechanisms involved. It is an invaluable companion for clinicians and scientists in all medical disciplines.
This unique volume in our Drugs and Pharmaceutical Sciences series covers the development of gene therapy today, the technology involved, clinical applications of siRNA, non-viral vector-based mRNA delivery using nanotechnology, and RNA based vaccines for treating the infectious diseases. It also presents the current application of the CRISPR/Cas9 gene-editing technique which has revolutionized genome editing and which was awarded the 2020 Nobel Prize in Chemistry. Several new drug delivery systems are explored for the applications of gene therapy. These are found to be useful in treating chronic illnesses, including cancer and infectious diseases. Key Features: Overview of the development of gene therapy Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing Presents CRISPR gene therapy recent trends and applications Discusses siRNA, mRNA, and DNA plasmids
Given the divergence in health policy between England and Wales, and the significant number of patients who cross the border for treatment, the Welsh Affairs Committee examined the interface between the two systems and the effectiveness of co-ordination between the Department for Health and the Welsh Assembly Government. It wanted to discover whether cross-border patients are treated fairly and whether the Welsh Assembly Government and the Department of Health consider the border in the development of the diverging policy environment. The Committee was aware of significant confusion amongst patients, for example in knowing what they are entitled to receive from their health service and that ...
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A listing of medical practitioners registered with the General Medical Council. Includes England, Scotland, Wales, and Northern Ireland. Data includes name, address, degrees, colleges, appointment, memberships, and publications. Also contains information on United Kingdom hospitals, NHS trusts, and boards of health.