• The issue of translatability of pre-clinical results was discussed with a wide range of stakeholders.

  • Challenges and pitfalls regarding the planning, funding, reporting and publishing of efficacy studies were discussed.

  • Future common efforts were planned to improve translatability of pre-clinical research for neuromuscular disorders.


Twenty-seven representatives of scientific journals, funding agencies, industry and academia from seven different countries (USA, Italy, Switzerland, Netherlands, Belgium, United Kingdom, Australia), and patient representatives met in Heemskerk in the Netherlands, on the weekend of the 10th–11th of February 2017 to help finalize a plan to guarantee quality in translational research for neuromuscular diseases. This workshop was the first to bring together a wide range of stakeholders to discuss the important issue of translational efficiency. The workshop is the fruit of the constant efforts of the TREAT-NMD Alliance to improve animal model studies for neuromuscular disorders with the final goal of accelerating research and effective treatment development.

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