NL
DE
Research
Rare and genetic movement disorders
About
We conduct clinical and translational research on specific groups of rare movement disorders, with a focus on ataxias. Central themes are: identification of new (genetic) causes; trial readiness; understanding disease mechanisms; and testing symptomatic and disease-modifying interventions.
Aims
For a prioritized set of rare movements disorders, we aim to:
- Secure trial-readiness, through natural history and biomarker discovery studies that provide sensitive and relevant outcome measures and inform the design of clinical trials;
- Identify new causes of rare movement disorders and implement these in diagnostic trajectories
- Generate new insights in molecular and system-level mechanisms, which serve as new disease markers or as leads for therapy;
- Design and test symptomatic and disease-modifying therapies.
