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 ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent beta-thalassemia ... involve collecting blood-forming stem cells from ...

obstructing smooth blood flow. Commonly known types of SCD include Haemoglobin SS, Haemoglobin SC, and Haemoglobin beta ...