Résumé :
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Promising therapies for neuromuscular diseases have been identified from many research areas and clinical trials for some of these therapies have begun. These long awaited developments have illustrated the lack of trial readiness amongst the neuromuscular community: a problem acknowledged by the EU in issuing a call for a Network of Excellence to work towards the acceleration of cutting edge therapies for the rare inherited neuromuscular diseases. Translational Research Europe- Assessment and Treatment of NeuroMuscular Diseases (TREAT-NMD) was commissioned by the EU for five years in January 2007 and 21 partners including industry and patient representation as well as academia and clinicians are involved in a series of activities directly addressing the pathway from the laboratory to the clinic in inherited neuromuscular diseases. The initial focus of the network has been Duchenne muscular dystrophy and spinal muscular atrophy but as the tools of the workshop develop, other target disorders are also being identified. Addressing the issues hindering the development of new therapies, initial targets of the network at the preclinical level have been the review of available animal models and generation of standardised operating procedures for their assessment. Issues around the delivery of therapeutic agents, techniques needed for their production and assessment of toxicology have been addressed and gaps in the current portfolio identified. Thinking about the delivery of clinical trials, outcome measure identification, and standards of patient care and diagnosis have been a prominent theme of the first year’s efforts, alongside the development of a harmonised global registry and clinical trial site identification. Active collaborations with other groups have resulted in a truly global perspective to the network and industry partnerships have been very promising. The EU asks for networks of excellence to be durable and sustainable which is a major challenge for years to come.
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