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

Project Details


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.
Effective start/end date1/01/0731/12/11


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