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

After a decade of relying on blood transfusions for her congenital disorder, 11-year-old Dang Thanh Mai Thu is now recovering ...

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 ...

Thalassemia is a blood disorder characterised by an increased concentration of hydrogen ions in the blood but can easily be prevented with a simple and inexpensive blood test. It is a genetic ...

There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: ...