4th Neuromuscular Translational School

Publicatie: 06 September 2022

The Neuromuscular Translational School organized by EURO-NMD and TREAT-NMD focuses on therapy development from bench to bedside with a focus on neuromuscular disorders. The Translational School contains lectures about the current state of the art of acquired and genetic neuromuscular diseases, the different steps of drug development and the tools needed for this (outcome measures, biomarkers etc). It also stresses how patients should play a role in each of these steps and teaches how to communicate research to patients and the general public during interactive workshop sessions.

The Translational School will take place in autumn this year from November 21-25 in Leiden University Medical Center, the Netherlands.

Target audience:

  • MDs
  • PhD/Postdoc researchers
  • Others working in translational research
  • Preferably in the NMD field, but in either case working in the RD field
  • Aim for 16 participants (definitely no more than 20 – or we lose the interactivity)
  • Industry delegates

Aim: To facilitate the clinical development of therapies for NMDs.


To educate clinicians and researchers working in the NMD field on aspects relevant for translational therapy development:

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

To outline and showcase how networks like EURO-NMD and TREAT-NMD facilitate therapy development:

  • Standards of care
  • Clinical trial tools
  • Outcome measure development
  • Interaction with stakeholders

Candidates are invited to send in their applications  via the ERN Euro-NMD website:


The programme can be downloaded here:

4th Neuromuscular Translational School Final Draft Program