Thalassemia is a common genetic condition affecting red blood cells. Children with severe forms of thalassemia often develop symptoms early in life, but effective treatment can manage the condition.

Whether you inherit alpha- or beta-thalassemia depends on which genes are affected. The number of affected genes can determine the subtype of alpha- or beta-thalassemia and the severity of your ...

Betibeglogene autotemcel gene therapy led to transfusion independence in 89% of patients with severe transfusion-dependent β-thalassemia. The trial involved 18 patients with specific genotypes, ...