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December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...
114,800 (2015) [8] Sickle cell disease ( SCD ), also simply called sickle cell, is a group of hemoglobin-related blood disorders typically inherited. [2] The most common type is known as sickle cell anemia. [2] It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2]
Sickle cell is the most common disease to receive approval for gene therapy treatment, following decades of development and years of approvals for therapy for people with rarer conditions.
By comparison, research earlier this year showed medical expenses for current sickle cell treatments, from birth to age 65, add up to about $1.6 million for women and $1.7 million for men.
Sickle cell disease consists of several subtypes; however, the Haemoglobin type C (HbSC) subtype carries the gravest prognosis for sickle cell retinopathy and vision changes. [2] Regular retinal examinations can aid in early detection and treatment, thus reducing the impact of the condition and the risk of vision loss.
Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.
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