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The company added that the data shows an imbalance in vaso-occlusive crises, a complication of the disease and "fatal events" that requires further as ... Pfizer withdraws sickle cell disease ...
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. [7]
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. [1][2] It is a form of sickle cell crisis. Sickle cell anemia – most common in those of African, Hispanic, and Mediterranean origin – leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries. The most common ...
Medical uses Lovotibeglogene autotemcel is indicated for the treatment of people aged twelve years of age and older with sickle cell disease and a history of vaso-occlusive events. [1][3]
The FDA approved a new treatment for sickle cell disease. The therapy is first to use the ground-editing tool CRISPR.
The FDA is reviewing a new drug for sickle cell disease that uses CRISPR to edit DNA. Black patients with the disease are excited and hopeful about the treatment.
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