Actualités

Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease (SCD) who are 12 or older. The company plans to submit the MAA by mid-2021. “Currently, there are no approved ...

  Patients with haemoglobin disorders, particularly β-thalassaemia or sickle cell disease (SCD) or combined forms, on account of their underlying disease pathology and associated (iron load mainly in the case of thalassaemia) co-morbidities, are defined as high-risk individuals, prone to develop more severe complications from SARS-COV-2. Despite the fact that ...

  IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia. The FDA had previously granted Orphan Drug Designation for IMR-687 for the treatment of ...

Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells. One of the novel therapies  for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion ...

Reblozyl’s long-awaited approval in Europe has finally arrived! Bristol Myers Squibb (BMS) & Acceleron Pharma announced today that the European Commission approved Reblozyl (luspatercept) for the treatment of Transfusion-Dependent β-#Thalassaemia (TDT) in adult patients. Reblozyl has the potential to address the ineffective erythropoiesis associated with β-thalassaemia and MDS, decrease patients’ dependence ...

  TIF was excited to participate in the 25th European Hematology Association (EHA) Annual  Virtual Congress, held this year virtually on 11 – 21 June 2020. Dr Androulla Eleftheriou (TIF Executive Director) and Ms Lily Cannon (TIF Operations Manager) have attended the following satellite symposia on 11 June: MDS AND ...

See what patients from various countries across the world who successfully took TIF‘s Thal e-Course, the ultimate self-paced, interactive, online Εducational Course for individuals with thalassaemia and their families, have to say about their experience with the course in their own language! Well-informed patients are able to gain in-depth understanding of their ...

TIF, a worldwide organization dedicated to ensuring equal access to quality healthcare for every patient with thalassaemia and other haemoglobin disorders across the world, and Hemanext Inc., a privately held medical technology company dedicated to improving patients’ quality of life by delivering a better red blood cell (RBC) replacement therapy, ...

A few days before the celebration of the World Blood Donor Day 2020, TIF has launched an online petition, co-signed by the European Blood Alliance (EBA), to promote  »Equitable Access to Safe and Adequate Blood » and voluntary, non-remunerated Blood Donation! Transfusion of blood and blood products help save millions of ...

Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class pyruvate kinase-R (PKR) activator Mitapivat for the treatment of patients with NTDT α- and β-thalassaemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety ...