At the Emma Center for Personalized Medicine, our mission is to unravel genetic underpinnings, establish preclinical models, and develop effective treatment for rare (genetic) disorders. We focus on different types of disorders, each with a genetic or metabolic cause and a high need for tailored treatments, paving the way for other disorders.

The selection of our focus diseases is driven by the availability of clinical cohorts, access to genetic data, biomarkers, and the feasibility of drug targeting. Additionally, the availability of resources, including EU platforms, enhances collaboration and accelerates progress.

We invite researchers, patients, and stakeholders to join our journey toward personalized therapeutic solutions, with the goal of improving outcomes and shaping a brighter future.

We use the therapy-accelerating wheel for every focus disease to accelerate therapy development. For more information on the Emma CPM wheel, click here.