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This Friday, 23 October 2020, at 15:00 EEST/13:00 BST, get ready to join Prof. Malcolm Walker and Dr. Dimitris Farmakis in our webinar on “Heart Disease In Thalassaemia“ for Patients/Parents,…

Imara Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of haemoglobin,  announced dosing of the first patient…

Johnson & Johnson has paused its Covid-19 vaccine trial due to an “unexplained illness” in a participant, the company confirmed yesterday evening. The pharmaceutical giant was unclear if the patient…

As public healthcare resources are redirected to halt the spread of coronavirus, victims of other life-threatening diseases pay the ultimate price. This recent news story, published by The Telegraph, depicts…

Fulcrum Therapeutics, Inc.  a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and…

As part of its enduring efforts to develop useful tools for individuals with thalassaemia and haemoglobin disorders worldwide, TIF will soon introduce a novel mobile health application, named ‘’THALIA Mobile…

Although chronic blood transfusion regimens have added decades to the lives of patients with thalassaemia, clinicians are now faced with increasingly complicated management challenges. The devastating effect of the accumulated…

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…

Bristol Myers Squibb Canada (BMS) and Acceleron Pharma Inc. announced today that Health Canada has approved REBLOZYL® (luspatercept) for the treatment of adult patients with red blood cell (RBC) transfusion-dependent anemia…