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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 ...
Because of a gene mutation, sickle cell anemia causes those red blood cells to break down early — taking a sickle or c-shape that carries less oxygen. It can lead to blocked blood flow, pain ...
The drug, called exa-cel, treats sickle cell disease, an inherited blood disorder that affects an estimated 100,000 people in the U.S., most of whom are Black, according to the Centers for Disease ...
DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
Samuel Charache. Samuel Charache (January 12, 1930 – January 29, 2019) was an American hematologist and professor at Johns Hopkins University. He led the research team that discovered the first effective treatment for sickle cell disease, a painful and sometimes fatal blood disorder that mainly affects people of African ancestry.
Victoria Gray. Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1] This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia. [1]
The two gene therapies are the first approved in the U.S. for sickle cell disease. The FDA has previously OK’d 15 gene therapies for other conditions. In the U.S., an estimated 100,000 people ...
Voxelotor. Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. [1][3][4][5][6] Voxelotor is the first hemoglobin oxygen-affinity modulator. [7] Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell ...
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