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  Global Blood Therapeutics, Inc. announced only a few hours ago that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta™ (previously known as Voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Oxbryta, an oral ...

  The World Health Organisation (WHO) has contacted the Thalassaemia International Federation (TIF) to request the provision of its expertise and technical support on the thalassaemia care and management of patients in the North West Syria region. Currently, this region includes four Thalassaemia Centres with approximately 500 patients. These patients ...

A new and encouraging chapter is finally opening up for patients with thalassaemia, following the recent approval of the US Food and Drug Administration (FDA) to market the drug Reblozyl, which aims to help patients with beta-thalassaemia major become more independent from regular, and often painful, blood transfusions. This important ...

  Celgene Corporation, Acceleron Pharma, Sangamo Therapeutics & Agios Pharmaceuticals are expected to present new and updated data on the progress of key ongoing therapies for thalassaemia and sickle cell disease (SCD) in the 61st American Society of Hematology Annual Meeting to take place in Orlando, Florida, 7-10 December 2019. ...

  The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product »Deferasirox Mylan», intended for the treatment of chronic iron overload due to blood transfusions in patients with β-thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias. The manufacturer of this medicinal product is Mylan S.A.S. »Deferasirox Mylan» will be ...

  The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale. The collaboration between the NIH and the Gates Foundation sets ...

Scientists have raised fresh hopes for treating people with genetic disorders by inventing a powerful new molecular tool that, in principle, can correct the vast majority of mutations that cause human genetic diseases. The procedure, named “prime editing”, can mend about 89% of the 75,000 or so harmful mutations known ...

  In a scientific paper recently published in Hematology, researchers discussed the most common pathophysiologic and clinical manifestations of renal disease in patients with beta thalassaemia. “In recent years, the life expectancy of patients with thalassaemia has increased, and this has allowed previously unrecognized renal complications to reveal themselves,” said ...

  Genome editing is a breakthrough healthcare technology which has the potential to notably change treatment options, largely influence current healthcare models, and impact patients in clinical trials worldwide. The science is advancing rapidly, promising considerable increases in the efficacy and precision of genome editing products with potentially curative treatments ...

On September 26, a Blood Transfusion Multi-Stakeholder Round Table Discussion was organised in Brussels by Celgene Corporation, featuring numerous scientists, haematologists, and patient organisations’ representatives. Dr. Androulla Eleftheriou, TIF Executive Director, and Mrs. Lily Cannon, TIF Operations Manager, represented the Federation in this meeting. The discussion drew attention to issues ...