What is beta-thalassemia?
Beta-thalassemia is a genetic disease that's caused by a change (or mutation) in the beta-globin gene. This mutation means your body cannot make enough of the beta-globin protein, which is a part of hemoglobin.
Having low or no beta-globin means you produce low amounts of adult hemoglobin. Not having enough hemoglobin in your red blood cells is why you might depend on transfusions.
Mutations in the beta-globin gene cause a lack of beta-globin production
Having low or no beta-globin means you produce low amounts of adult hemoglobin (HbA)
Without enough adult hemoglobin, red blood cells do not function normally
Without adult hemoglobin, red blood cells are unable to carry oxygen throughout the body
The need for increasing hemoglobin.
Healthy red blood cell
Beta-thalassemia red blood cell
Red blood cells will have a shorter life span without hemoglobin, and will carry less oxygen throughout the body causing anemia. If you’re not able to produce sufficient hemoglobin, you may need regular transfusions.
Regular transfusions address the symptoms of beta-thalassemia, but do not treat it at the genetic level.Browse FAQs
The switch from fetal hemoglobin to adult hemoglobin.
You may have previously heard of fetal hemoglobin. This is a type of hemoglobin that makes up most of the total hemoglobin while a body is developing as a fetus. Shortly after birth, your body will naturally switch to producing mostly (over 90%) adult hemoglobin as part of typical normal development.
Talk to your doctor about ZYNTEGLO
Your doctor and care team can help determine whether ZYNTEGLO may be an appropriate treatment option for you.
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