In the field of rare diseases it is extremely difficult to perform clinical trials with new chemical entities without prior information on bioavailability, dose and safety in humans. Drug repurposing is the application of known drugs and compounds to new indications. Thus, the aim of this project is to repurpose existing medicines to treat Fanconi anemia. This long term effort implies the development and use of disease-specific cell based systems to screen beneficial effects of known drugs.
Researchers: Jordi Surralles