Patients

Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of…

With open dialogue-formatted episodes, featuring thalassaemia patient advocates, mental health professionals, and other special guests, this show explores everything from psychosocial anxiety and relationship issues to treatment-related fears, while striving…

This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…

Physicians in Norway have initiated recruitment of chronically transfused patients with haematological malignancies, including myelodysplastic syndrome (MDS), to receive transfusions using RBCs processed and stored with the Hemanext ONE® system.…

Every single one of us has the ability to make a difference, large or small, and together we can make real progress in raising valuable awareness about thalassaemia and its…

The objective of this meeting, which brings together eminent haemoglobinopathies specialists and expert patient advocates from Italy and other countries, is to highlight the pivotal value of advocacy in enabling…

Where was my wife? I found myself sitting alone in front of the obstetrician, who my wife, Helen, and I had met for the first time only minutes earlier. But…

Rare Disease Day is the movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease everywhere…