Queen Square Centre for Neuromuscular Diseases



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 standards.

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 scientists, including the group of students on the doctoral training programme, in patient-centred clinical and scientific activities is also a priority.

Basic 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 underdeveloped.

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 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.