The first-in-class erythroid maturation agent luspatercept (Reblozyl) is already approved for the treatment of anemia in adult patients with beta thalassemia who require red blood cell (RBC) ...
Please provide your email address to receive an email when new articles are posted on . Thompson described beta-thalassemia as a severe chronic inherited blood disorder due to one of many mutations in ...
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that both pivotal trials for exagamglogene ...
Please provide your email address to receive an email when new articles are posted on . An investigational CRISPR/Cas9 gene-edited cell therapy demonstrated significant, durable efficacy among ...
Today new and updated data from bluebird bio inc.’ s gene therapy programs in beta-thalassemia and sickle cell disease were presented at the 64 th American Society of Hematology Annual Meeting and ...
SOMERVILLE, Mass.--(BUSINESS WIRE)--Following the FDA approval of ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, for the treatment of beta-thalassemia in adult and pediatric patients ...
An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...
A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial ...
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