In light of the rapid scientific developments in the field of thalassaemia and Sickle Cell Disease (SCD) – new drugs and gene therapies – which promise to change the course of these diseases, and given the leading global role of the Thalassaemia International Federation (TIF) since 1986 in informing ...
Read More »FDA Approves Oxbryta™ (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease
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 ...
Read More »TIF by the side of thalassaemia patients in Syria
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 ...
Read More »TIF will coordinate efforts for patients’ information on Reblozyl in 62 countries
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 ...
Read More »New and Updated Data on Key Thalassaemia & SCD Pipeline Therapies at the American Society of Hematology (ASH) 2019 Annual Meeting – 7-10 December 2019
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. ...
Read More »Deferasirox Mylan: A new generic drug for the treatment of iron overload in β-thalassaemia major obtained approval by the EMA
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 ...
Read More »US government & Gates Foundation pledge $200 million to bring gene therapies to patients with Sickle Cell Disease & HIV
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 ...
Read More »New CRISPR editing tool could potentially fix 89% of genetic variations in human diseases
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 ...
Read More »High-Level Thalassaemia & Sickle Cell Disease Healthcare Professionals Summit – 1 & 2 November 2019
After the successful Capacity Building Workshop for Patient Organizations & Patients with thalassaemia in Hamburg, Germany, TIF will be traveling to Thessaloniki, Greece for a High-Level Thalassaemia & Sickle Cell Disease Healthcare Professionals’ Summit, to be held on 1-2 November 2019. Seeking to inform the healthcare community working in Europe ...
Read More »As survival increases in β-thalassemia, renal complications emerge
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 ...
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