The translational research environment enables a clear overarching goal to translate molecular discovery
into benefits for patients and is ideal for training the clinical and
basic scientists of the future.
It also represents a UK national
centre of excellence offering training opportunities and setting
It provides the largest clinical neuromuscular services for children and adults in 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 is a major focus of the centre in order to generate new future capacity.
Training basic scientists
Translating molecular discoveries into patient benefits runs through all training and educational activities undertaken in the centre.
The often neglected involvement of basic
the group of students on the doctoral training programme, in patient
centred clinical and scientific activities is also a priority.
scientists have regular exposure to the clinical
problems, phenotypes and complications of neuromuscular disease
patients. This is through attendance at neuropathology conferences,
clinical-pathological meetings, "bedside to bench" seminars as well as
clinical research seminars.
In this way all science students 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 and by reinforcing this linkage, we hope to help 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
There is 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 pessimistic approach to therapeutic
options for chronic neuromuscular disease.
In both paediatric and adult
practice it is now recognised that appropriate management and
therapeutic intervention of hereditary neuromuscular disease has
significantly improved quality and duration of life of affected
Examples are the intervention related to respiratory and cardiac complications for neuromuscular diseases, and the use of pharmacological intervention using corticosteroids in muscular dystrophy.