While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell ...

However, the risk of complications associated with the treatment of thalassemia will change as children transition into ...

Thalassemia is a hereditary blood disorder causing anaemia due to reduced haemoglobin production. Early detection, awareness, ...

For beta thalassemia, the part of the hemoglobin ... With a mild case, you may feel tired and not need treatment. But if it's more serious, your organs may not get the oxygen they need.

A family of Albanian descent had an unusual form of beta thalassemia. The father of two children with mild thalassemia major had normal red-cell morphology and normal levels of hemoglobins A 2 and ...

“Our best-in-class gene therapy has the potential to cure patients living with beta thalassemia and sickle cell disease ...

ENERGIZE-T Study Achieved Primary and All Key Secondary Endpoints in Adult Patients with Transfusion-Dependent Alpha- or Beta-Thalassemia - - ENERGIZE-T is First Phase 3 Study to Demonstrate Efficacy ...

In June 2024, Agios also presented positive results from the Phase 3 ENERGIZE study, which evaluated mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia. “Treatment ...