An application for the ERN in Rare Hematological diseases (ERN-RHD) is being developed led by ENERCA coordinator, Prof Joan Lluis Vives Corrons.
Network disease coverage will be expanded by including Rare Hematological diseases in thematic sub-nets.
JOIN THE ERN-RHD (here)
May 17, 2016
EURORDIS has developed ePAGs for each ERN disease grouping. ePAGs bring together elected patient representatives and affiliated organisations who will ensure that the patient voice is heard throughout the ERN decision-making processes and a democratic process of patient representation.
May 9, 2016
The Food and Drug Administration (FDA), the govern agency of United States responsible for the drugs regulations, has designated “lentiviral vector containing human liver and erythroid pyruvate kinase (PKLR) gene” as new Orphan Drug for the PKD treatment, a rare anaemia caused by mutations in the gene that codifies the pyruvate kinase enzyme, which impairs directly the metabolic energy of red cells and results in the reduced lifespan and early breakdown of erythrocytes in the blood.
June 6-10, 2016
June 9-12, 2016