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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.
December 8, 2023 at 10:19 AM. 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 ...
The Food and Drug Administration says there’s an unmet need for help for patients suffering from severe sickle cell anemia. A new drug that uses gene editing could solve that.
146 232.04 g·mol −1. Crizanlizumab, sold under the brand name Adakveo among others, is a monoclonal antibody medication that binds to P-selectin. [3] It is a medication used to reduce the frequency of vaso-occlusive crisis in people aged 16 years and older who have sickle cell anemia. [3][4][6] It is given by injection into a vein. [3][4 ...
A Mississippi woman was the first patient to test a treatment that may be a new cure for sickle cell disease. ... If the treatment is allowed on the market, the company has proposed a post ...
As disclosed on October 22, the Company established in vivo preclinical proof of concept for the development of an in vivo medicine for sickle cell disease and beta thalassemia by demonstrating in vivo hematopoietic stem and progenitor cell (HSPC) editing of HBG1/2 promoter utilizing a novel, Editas-proprietary targeted lipid nanoparticle (tLNP ...
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 ...
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
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