One of the main interests of EPTRI includes the rare diseases (RDs) as they affect mainly children, and genetic RDs start early in the prenatal/childhood life with a high frequent use of medicines not specifically tested (off-label, unlicensed). To this aim, EPTRI recently became a friend of Rare Disease Day, an initiative organised by EURORDIS that takes place on the last day of February each year. The aim of this day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.
EPTRI fully embraces the goals of this important initiative that will be glad to promote among its partners, supporters and stakeholders, spreading the event on its website and social channels.
EPTRI can make the difference in closing the gap between innovative technologies and will work to accelerate the paediatric drug development processes from medicines discovery, biomarkers identification and preclinical research to developmental pharmacology, age tailored formulations and medical devices.