Understanding Thalassemia

What is thalassemia?

Thalassemia is a blood disorder caused by inherited mutations in the alpha- or beta-globin genes. As a result, the body is not able to make enough hemoglobin, an important part of red blood cells. People with alpha- and beta-thalassemia can experience a range of symptoms, including anemia, debilitating fatigue, jaundice, facial bone deformities, delayed growth and development, abdominal swelling and dark urine.

There are no currently approved therapies for alpha-thalassemia and limited options for beta-thalassemia. Current management strategies for beta-thalassemia include ​​red blood cell transfusions, splenectomy and stem cell transplant, which are associated with short- and long-term risks.

Find out more about our clinical trials in thalassemia and talk to your doctor if you’re interested in participating in studies that are currently recruiting.

Thalassemia Newsletter

Connecting the dots in thalassemia

Thalassemia is an inherited blood disorder caused by mutations in either alpha- or beta-globin genes, reducing the production of functional hemoglobin, the protein in red blood cells that carries oxygen. This causes a shortage of red blood cells and low levels of oxygen in the bloodstream, leading to a variety of health problems.

Learn more about our pioneering approach and scientific expertise in cellular metabolism.

A pipeline of possibility

Building on our core capabilities in cellular metabolism, we are advancing a robust pipeline focused on rare diseases, including thalassemia.

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Connected to patients

At the end of the day, we are creating therapies for patients. As the true experts in their disease, their needs, concerns, input and collaboration are critical to our work.

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