Mitapivat improves clinical outcomes in patients with non-transfusion dependent thalassemia
1. Hemoglobin response was significantly greater in the treatment group compared to placebo. 2. Majority of adverse events were mild ...
Tag: β-thalassemia
Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia
1. 89% of patients achieved transfusion independence with beti-cel gene therapy. 2. There were no serious adverse events or treatment-related ...
#VisualAbstract: Deferoxamine, Deferasirox, and Deferiprone Triple Iron Chelator Combination Therapy for β-Thalassaemia
Click here to read this study in the Lancet. ©2024 2 Minute Medicine, Inc. All rights reserved. No works may be ...
Mitapivat may improve anemia in adults with non-transfusion dependent α- and β-thalassemia
1. 80% of patients receiving mitapivat showed hemoglobin response at 12 weeks. 2. Markers of hemolysis, such as bilirubin and ...
Betibeglogene autotemcel gene therapy effective for β-thalassemia
1. Betibeglogene autotemcel (beti-cel) gene therapy resulted in sustained hemoglobin A with a T87Q amino acid substitution (HbAT87Q) in non-β0/β0 ...
