Research Research programs Therapy development for rare disorders of the brain

Our mission

We aim to bring new, effective and affordable therapies to patients with rare disorders of the brain.


Goals

See some of the most important goals for this research program.

read more

Goals

  • To establish preclinical model systems with predictive validity for candidate interventions. 
  • To secure access to both disease-specific and generic clinical and surrogate markers that can serve as sensitive and patient-relevant read-outs in clinical trials. 
  • To design and conduct academic-led clinical n-of-1 or n-of-few trials for lead compounds. 


Research groups contributing to this research program

Spokesperson(s) for this research program


Spokesperson

prof. dr. Bart van de Warrenburg

contact form

Spokesperson

prof. dr. Dirk Lefeber

contact form