The second Neuromuscular Translational Summer School built on the success of the first, and took place in Leiden, The Netherlands on 1st – 5th July 2019.
The five-day course which was organised and delivered in association with TREAT-NMD 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.
Download our 2019 summer school programme at a glance.
We are in the process of developing our programme for the next summer school which will be held in 2020. Further information will be available in due course please regularly visit the website or monitor our twitter feed to ensure you keep up to date.