Personalized care in the right place for patients with genetic disorders. Once therapies have been identified, applied and evaluated, we aim to ensure that therapeutic possibilities are implemented in clinical care, for both the current patient and future once. To optimize healthcare evaluation, patients are actively involved in assessing and monitoring their individual treatment responses based on endpoints that are relevant to them.

Patients benefit from a “one-stop shop” approach, which is a single visit to a specialized multidisciplinary team instead of multiple outpatient appointments with different healthcare providers at different times.

We are developing new care pathways to ensure that patients receive the right care in the right place, minimizing the burden on both patients and their families. These new care pathways form the foundation for recognition as Centers of Expertise for Rare Diseases (ECZA) by the Ministry of Health, Welfare, and Sport (VWS).