TREAT-NMD
Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world
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6th Neuromuscular Translational Summer School
9 July 2024 - 12 July 2024
The 6th 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 Summer School contains lectures about the current state of the art of the management 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.
Aims and objectives
- Facilitate the clinical development of therapies for NMDs
- 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
- 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
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