Therapeutic areas


Sickle cell anemia, also known as sickle cell disease, is a hereditary genetic disease caused by a mutation in gene encoding hemoglobin, the molecule in red blood cells that transports oxygen from the lungs to other parts of the body.

Red blood cells with normal hemoglobin (hemoglobin-A) are smooth, round and flexible therefore, they glide through blood vessels, even in tight capillary beds. People with sickle cell anemia have abnormal hemoglobin molecules (hemoglobin-S) that can form long, rod-like structures and clump together. This causes red blood cells to become stiff, assuming a sickle shape. These sickled red blood cells tend to pile together, creating clots, and blocking the flow of blood through vessels. This can cause a number of complications, including vaso-occlusive crises with damage to several organs and disabling pain episodes.

Problems due to sickle cell anemia can begin at a very young age1. Therefore, an early and adapted management of the disease in children is crucial to minimize the impact of these complications.