Non-transfusion-dependent thalassemia encompasses three clinically distinct forms -- beta-thalassaemia intermedia, hemoglobin E/beta-thalassaemia (mild and moderate forms), and al ...

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

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: ...