Sickle cell thalassemia occurs when you inherit both a sickle cell gene mutation and a thalassemia gene mutation. Symptoms are similar to other types of sickle cell disease but can vary depending on ...

People with the thalassemia trait, or thalassemia carriers, may not experience any symptoms. However, some thalassemia carriers can experience anemia symptoms, such as paleness and fatigue. In people ...

While mild anemia is generally treatable, some severe forms, like thalassemia and aplastic anemia, can lead to life-threatening complications if untreated due to the body’s organs not receiving enough ...

An ongoing, phase 3, open-label study demonstrated the efficacy of exagamglogene autotemcel (exa-cel) infusion to promote transfusion dependence in patients with transfusion-dependent β-thalassemia ...

CLEVELAND, Ohio -- Cleveland Clinic Children’s Hospital is among the first in the country to use a new gene therapy to treat patients with thalassemia, a genetic blood disorder that affects the ...

About PYRUKYND® (mitapivat) U.S. INDICATION PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.