TREAT-NMD / EURO-NMD Neuromuscular Translational Summer Schools: showing all the dimensions of neuromuscular diseases

The first Neuromuscular Translational Summer School took place in July 2018 in Newcastle upon Tyne, UK. The Summer School was the result of a collaborative effort between TREAT-NMD and EURO-NMD to address specific aspects of the established translational pathway in clinical trials. Fifteen attendees from different backgrounds within the neuromuscular community took part in the Summer School. They included clinicians, researchers, patient registry curators and representatives from the pharmaceutical industry. The 17 faculty members were a mixture of multi-disciplinary clinicians, patient representatives, a regulatory body representative, behavioural scientists and professional services staff.

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The summer school aimed to provide participants with a foundation level knowledge of the translational lifecycle, clinical trials, biomarkers and patient communication from ‘Bench to Bedside’. Feedback from attendees was very positive. Participants particularly liked the informal and interactive nature of the programme and sessions and the expertise of the presenters.  

The second Neuromuscular Translational Summer School built on the success of the first, and took place in Leiden, The Netherlands on 1^st – 5^th July 2019. The five-day course included an overview of the following:

• Neuromuscular diseases and current care and management practices.
• Bench to bedside research.
• Challenges for rare disease therapy development and networking solutions.
• Tools of the trade for preclinical research.
• An introduction to TACT (TREAT-NMD Advisory Committee for Therapeutics) and a TACT mock review session.
• Introduction to clinical trials.
• How the regulatory system works.
• The Industry perspective on drug development for rare diseases.
• Outcome measures used in clinical trials.
• TREAT-NMD tools for clinical trials.
• Biomarkers.
• PROM development.
• Post-marketing.
• Patient engagement.

The 3^rd Summer School will be held in 2020 in Leiden. Dates will be announced soon!

More information about the Summer Schools is available on the EURO-NMD website. Feedback from a participant of the 2019 Summer School is available in the July 2019 TREAT-NMD newsletter.