Researchers at Newcastle University are leading the 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.”
Claire Jordan | alfa
Researchers release the brakes on the immune system
18.10.2017 | Rheinische Friedrich-Wilhelms-Universität Bonn
Norovirus evades immune system by hiding out in rare gut cells
12.10.2017 | University of Pennsylvania School of Medicine
University of Maryland researchers contribute to historic detection of gravitational waves and light created by event
On August 17, 2017, at 12:41:04 UTC, scientists made the first direct observation of a merger between two neutron stars--the dense, collapsed cores that remain...
Seven new papers describe the first-ever detection of light from a gravitational wave source. The event, caused by two neutron stars colliding and merging together, was dubbed GW170817 because it sent ripples through space-time that reached Earth on 2017 August 17. Around the world, hundreds of excited astronomers mobilized quickly and were able to observe the event using numerous telescopes, providing a wealth of new data.
Previous detections of gravitational waves have all involved the merger of two black holes, a feat that won the 2017 Nobel Prize in Physics earlier this month....
Material defects in end products can quickly result in failures in many areas of industry, and have a massive impact on the safe use of their products. This is why, in the field of quality assurance, intelligent, nondestructive sensor systems play a key role. They allow testing components and parts in a rapid and cost-efficient manner without destroying the actual product or changing its surface. Experts from the Fraunhofer IZFP in Saarbrücken will be presenting two exhibits at the Blechexpo in Stuttgart from 7–10 November 2017 that allow fast, reliable, and automated characterization of materials and detection of defects (Hall 5, Booth 5306).
When quality testing uses time-consuming destructive test methods, it can result in enormous costs due to damaging or destroying the products. And given that...
Using a new cooling technique MPQ scientists succeed at observing collisions in a dense beam of cold and slow dipolar molecules.
How do chemical reactions proceed at extremely low temperatures? The answer requires the investigation of molecular samples that are cold, dense, and slow at...
Scientists from the Max Planck Institute of Quantum Optics, using high precision laser spectroscopy of atomic hydrogen, confirm the surprisingly small value of the proton radius determined from muonic hydrogen.
It was one of the breakthroughs of the year 2010: Laser spectroscopy of muonic hydrogen resulted in a value for the proton charge radius that was significantly...
17.10.2017 | Event News
10.10.2017 | Event News
10.10.2017 | Event News
20.10.2017 | Information Technology
20.10.2017 | Materials Sciences
20.10.2017 | Interdisciplinary Research