

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