Sickle Cell Disease (SCD) is an inherited disease that affects the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. The disease occurs when people inherit a mutation from each of their parents. This mutation causes people with SCD to not have normal, healthy adult hemoglobin in their red blood cells and instead have an abnormal hemoglobin called sickle hemoglobin. The result is rigid, crescent-shaped, or “sickled” red blood cells that can clump together in blood vessels and block the flow of blood throughout the body. Consequently, SCD can cause frequent episodes of severe pain, weakness and other serious complications.
The Importance of Fetal Hemoglobin
Fetal hemoglobin is an “anti-sickling” hemoglobin that is present before birth in the red blood cells. After birth, the gene that makes fetal hemoglobin turns off, which mostly stops the production of fetal hemoglobin. More fetal hemoglobin in the blood can mean fewer episodes of sickling and pain.
The MOMENTUM study is investigating a one-time investigational treatment to increase levels of fetal hemoglobin in patients with severe sickle cell disease, with the hope of fewer episodes of sickling and pain.