Κλινικά Νέα

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…

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.…

  The National Institutes of Health’s (NIH) work on a universal flu vaccine is already well advanced, with human trials beginning now. The new formulation, called BPL-1357, contains a variety…

The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…

Many pharmaceutical companies are making remarkable progress in their research into candidate treatments for these diseases and 2022 is expected to be yet another eventful year for haemoglobin disorders! Check…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic…

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…

Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged…

A key objective of the Thalassaemia International Federation is to inform and update regularly the global haemoglobinopathies family, both patients and physicians, on the progress and new developments that arise…

For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK National Health Service (NHS). Crizanlizumab (Adakveo) by Novartis…