The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Insertion One or more extra nucleotides are inserted into replicating DNA, often resulting in a frameshift One form of beta-thalassemia ... Inversion One region of a chromosome is flipped and ...

You most likely have beta thalassemia minor (one normal gene and one that causes low beta-cell-chain production), which consists of mild anemia and very small cells. This condition can look very ...

Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered ...