Sickle Cell Disease

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…

bluebird bio, Inc. today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin…

Global Blood Therapeutics, Inc. announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study of Oxbryta® (voxelotor) tablets in patients with sickle cell disease…

Hemanext Inc. announced today it has received a CE Certificate of Conformity for the CE Mark for its Hemanext ONE Red Blood Cell (RBC) Processing and Storage System, allowing the…

TIF is delighted to announce the organisation of a Virtual Presentation Event of the ‘TIF Sickle Cell Disease Educational Course for Healthcare Professionals’ – an online course providing comprehensive information…

TIF joins forces with 300 million people around the world for the global observance of Rare Disease Day, coming up on February 28th. Rare Disease Day is an annual awareness…

The Thalassaemia International Federation (TIF) has been informed through official communication by both the European Medicines Agency (EMA) and bluebird bio that the latter has placed Phase 1/2 and Phase…

The European Medicines Agency (EMA) has accepted Global Blood Therapeutics (GBT) marketing authorisation application (MAA) for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia in sickle cell disease (SCD). According to the company,…

The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…