Abstract
In June 2014 TREAT-NMD organised a workshop at The Wellcome Trust head office in London to discuss issues such as natural history, outcome measures, efficacy measurements, animal models and clinical study design as part of the coordinated response to the public consultation by the European Medicines Agency (EMA) on the draft guidelines for clinical investigation of medicinal products in Duchenne and Becker muscular dystrophy (DMD/BMD). The workshop attracted over 60 participants from 9 countries representing patients, academics, and industry.
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