Sickle cell
disease
Vertex is focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
Vertex is focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
What is sickle cell disease?
Sickle cell disease (SCD) is a serious, inherited blood disorder. It affects the red blood cells (erythrocytes), which are essential for transporting oxygen to all organs and tissues in the body. SCD occurs due to a mutation in the so-called beta globin gene (HBB). The mutation leads to the formation of the so-called sickle haemoglobin (HbS). Due to HbS, red blood cells change after oxygen is released and take the shape of a sickle, which gives the disease its name.
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It is estimated that 17,500 people in the UK have sickle cell disorder.1
How is SCD diagnosed? In Europe, most newborns are screened for SCD at birth, while symptoms usually take about 6 months to present.
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The Sickle Cell Society. About Sickle Cell. Last accessed May 2024