Translational School on Neuromuscular Diseases

Our annual winter school, which in association with TREAT-NMD, is especially developed for researchers and clinicians interested in translational research.

The following topics will be covered:

-Bench to bedside research
-Regulatory system
-Clinical trials
-Outcome measures
-Patient communication
-Registries and biobanks
-Biomarkers and –omics

Target group

Researchers and clinicians interested in translational research in the neuromuscular disease space.

Course aim

Attendees of the winter school discover how networks such as the European Reference Network EURO-NMD and TREAT-NMD work and collaborate with patients and regulators to facilitate therapy development. Aspects such as standards of care, outcome measure development, biomarkers and others are discussed and debated during the week. Attendees also have the opportunity to find out about the various interactions that take place between key stakeholders how the regulatory system works and take part in a TACT style review and indepth discussion on ethical issues in clinical trials.