June 25, 2020 – The Thalassaemia International Federation’s (TIF’s) position statement on COVID-19 and Thalassemia has just been published in the European Journal of Haematology. You can access the position statement here: https://www.thalassemia.org/wp-content/uploads/2020/06/COVID-19-and-Thalassaemia-A-Position-Statement-of-the-Thalassaemia-Int….pdf The article notes that “The optimization of blood use will help safeguard blood supplies during the pandemic…One particular aspect of optimizing blood […]
June 22, 2020 – If you were unable to join us for last Thursday’s webinar entitled “Overview of Novel Thalassemia Treatments” by Dr. Sujit Sheth of the New York Presbyterian Hospital/Weill Cornell Medical Center, the archived webinar can now be accessed online at: https://www.cdc.gov/ncbddd/blooddisorders/webinar.html
June 16, 2020 – Dr. Maria-Domenica Cappellini of the University of Milan, Italy, recently presented the following data showing the outcomes of COVID-19 infection in a cohort of 11 patients with thalassemia from Northern Italy. These 11 Italian thalassemia patients experienced relatively mild to moderate COVID-19 disease. All the patients had thalassemia-related comorbidities. 53.5% of […]
June 12, 2020 – Bluebird bio has reported the findings from its Phase 3 clinical trial program of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia). These show that pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia are able to achieve and maintain transfusion independence with hemoglobin (Hgb) levels that are near-normal (≥10.5 […]
June 12, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal hemoglobin […]
