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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 ...
The price tags for the two sickle cell therapies in the U.S. are $3.1 million and $2.2 million although costs can vary by country. The process of giving the therapies is just as big a hurdle.
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.
D11930. Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [ 1 ][ 5 ][ 2 ] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015. [ 6 ][ 7 ] The most common adverse reactions include reduced ...
This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and Prevention shows crescent-shaped red blood cells from a sickle cell disease patient in 1972.
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. [ 1 ][ 2 ][ 3 ] The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans ...
Now, 33, Stephenson was one of the first to receive sickle cell gene therapy in a clinical trial six years ago. Before the treatment, Stephenson lived her life on a razor's edge.
Britain's regulator said its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at ...
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