TREAT-NMD Translational Research in Europe - Assessment and Treatment of Neuromuscular Diseases

TREAT-NMD is a European neuromuscular network addressing the fragmentation currently hindering translational research for cutting edge therapies in rare neuromuscular diseases (NMD). It will strengthen European excellence in the treatment of rare NMD. Bringing promising cutting edge therapies into clinical settings is currently delayed by the lack of standardised protocols for preclinical animal studies, molecular diagnoses and patient assessment and management. TREAT-NMD will address this fragmentation by establishing a common road map for the progression of cutting edge therapies from laboratory to clinic, from the assessment of cellular and animal models, via issues of delivery, production and toxicology, to clinical outcome measures. This will be underpinned by the integration and establishment of pan-European patient databases and biobanks. The TREAT-NMD coordination centre (TNCC) will develop and integrate organisations and networks comprising the top researchers, clinicians and industries working in Europe (representing 11 countries) in partnership with patient organisations in order to deliver the dream of treatments for these devastating disorders. Technological, educational and communication Centres of Excellence will be generated as a durable resource co-ordinated by the TNCC, which will establish Europe as the leader in translational research in this area. A strong educational programme will provide the next generation of experts with the necessary background and expertise to maintain this leading position, and integrate the voices of patient organisations in the development of future research plans. Cutting edge treatments currently under development for muscular dystrophies and spinal muscular atrophy will be specifically targeted in this process, while future developments will also benefit from the durable infrastructure of the TNCC, which will act as the portal for future pharmaceutical developments in this field