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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. 


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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.

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Research groups contributing to this research program

Bioinformatics

Peter-Bram 't Hoen's research group develops computational workflows for the integration of omics and clinical data to predict disease progression and identify drug repurposing candidates in myotonic dystrophy and other neuromuscular disorders. 

Cellular bioenergetics

Werner Koopman's research group focuses on understanding cellular energy metabolism in the context of the mitochondrial (dys)function at multiple levels (organisms, tissues, cells, proteins, molecules), with the aim to develop intervention strategies.

Clinical genetics of intellectual disability

Bert de Vries's research group focuses on the identification of novel genes involved in neurodevelopmental disorders such as intellectual disability and autism spectrum disorder, and the clinical characterization of those novel genetic syndromes.

Spokesperson(s) for this research program

Spokesperson

prof. dr. Bart van de Warrenburg

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Spokesperson

prof. dr. Dirk Lefeber

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