Researchers at Newcastle University are leading the 6.7m (10m euros) 'network of excellence', the first of its kind, which has been funded by the European Union following extensive lobbying from the European Organisation for Rare Diseases (EURORDIS), the French Muscular Dystrophy Association (AFM) and other patient groups and clinicians.
Called TREAT-NMD (stands for Translational Research in Europe - Assessment and Treatment of Neuromuscular Diseases), the network brings together 21 partner organisations, including doctors, researchers, charities and public companies already working on neuromuscular diseases, based throughout 11 European countries. The funding lasts for five years although network leaders hope it will survive beyond this timescale and expand to include other international partners.
Neuromuscular disorders affect around 200,000 people in Europe and up to 30,000 people in the UK. The term refers to a large group of conditions that affect either the muscles themselves, or the nerves controlling the muscles. Most conditions result in chronic long term disability and early death may eventually result from respiratory failure.
TREAT-NMD will encourage experts in this field to work together to share good practice and to improve global standards of care.
Scientists will work closely with doctors to test and apply new research into these inherited disorders, in order to develop new ways of looking after patients with conditions like Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA).
Close links with drug companies will also be further developed to advance potential treatments and cures. Patients and patient charities will be heavily involved at all levels.
Other special features of TREAT-NMD include the development of a clinical trials co-ordination centre in Germany, which will provide advice on how to conduct trials of the highest standard, and the option of top-level training for network members.
Kate Bushby and Volker Straub, Professors of Neuromuscular Genetics with Newcastle University's Institute of Human Genetics, who are based at Newcastle's Centre for Life, are co-ordinating TREAT-NMD. They are also practising doctors with the Newcastle upon Tyne Hospitals NHS Foundation Trust.
The network is launched 20 years after scientists found the gene that caused DMD but as yet no cure has been found.
Prof Bushby said: "At the moment no cures exist for these incredibly disabling diseases so patients receive treatment from doctors like me and Professor Straub to manage their condition in the best way.
"Standards of care and treatment differ greatly throughout the world but the level of treatment can make a great difference in terms of quality of life and life expectancy."
Prof Straub said: "It is a great honour that Newcastle was chosen to lead this international network of excellence. The application would not have been successful without the fantastic support we received from all of our European partners. The French Muscular Dystrophy Association was particularly helpful as they provided us with funds for the application process."
Domenico Marchetti, who had a son affected by Spinal Muscular Atrophy (SMA) and is president of SMA Europe, said: "SMA is the main genetic cause of death for children under the age of two. Tens of thousands of families, all over the world, fight every day with this terrible disease. TREAT-NMD is a great step forward along the path to improve children's quality of life now, and, tomorrow, to find a cure."
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