Teaching

Clear translational research training focus-“bench to bedside and back"

The translational research environment our centre will create with a clear overarching goal to translate molecular discovery into benefits for patients will be ideal for training the clinical and basic scientists of the future. It will also represent a UK national centre of excellence offering training opportunities and setting standards. The juxtaposition of “state of the art” basic science facilities [ION/ICH/UCL] with the largest clinical neuromuscular services for children and adults in the UK and indeed Europe, is truly unique, and provides limitless opportunities for the training and education of basic and clinical scientists.The training of clinical and non-clinical scientists in neuromuscular disease will be a key major focus of the centre in order to generate new capacity for the future.

Training basic scientists: the theme of translating molecular discoveries into patient benefits will run through all training and educational activities undertaken in the centre. The often neglected involvement of basic scientists, including the group of students on the doctoral training programme, in patient centered clinical and scientific activities will be a priority. Basic scientists will therefore have regular exposure to the clinical problems, phenotypes and complications of neuromuscular disease patients. This will be through attendance at neuropathology conferences, clinical-pathological meetings, “bedside to bench” seminars as well as clinical research seminars. In this way all science students will develop a clear understanding of the target neuromuscular disease their research is focused upon. We strongly believe this strong linkage between the patient disease and the basic science is often missing in doctoral training programmes. By reinforcing this linkage, we hope this will help to inspire talented young scientists to develop their career in neuromuscular research and become the leaders of the future.

Training clinical scientists: compared with other neurological diseases, the training of clinical scientists in neuromuscular disorders has been relatively neglected and underdeveloped. There is therefore a shortage of clinical scientists with the expertise needed to deliver molecular therapies and other clinical trials in this area. There are many reasons for this shortage including lack of centres of excellence which offer specific training programmes and also a previous nihilistic approach to therapeutic options for chronic neuromuscular disease. In both paediatric and adult practice it is now recognized that appropriate management and therapeutic intervention of hereditary neuromuscular disease has significantly improved quality and duration of life of affected individuals. Examples are the intervention related to respiratory and cardiac complications for neuromuscular diseases, and the use of pharmacological intervention using corticosteroids in muscular dystrophy.