Develop and patent a drug for the specific treatment of Diamond-Blackfan Anaemia

Diamond-Blackfan anaemia (DBA) is an inherited chronic disease in which patients suffer from severe anaemia. There is at present no treatment that specifically corrects the cause of the anaemia. Instead, patients are treated from infancy with blood transfusions or corticosteroids, which over time, leads to serious side effects and premature death. The goal of the project is therefore to develop a specific drug that corrects the underlying problem for this group of patients.