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Sickle cell-beta thalassemia is an inherited blood disorder. The disease may range in severity from being relatively benign and like sickle cell trait to being similar to sickle cell disease . [ 1 ] [ 2 ]
Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [3] [5] and transfusion-dependent beta thalassemia. [3] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. [9]
The groundbreaking treatment can now also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder.
A gene-editing therapy for sickle cell disease, with a price tag of £1.65m, is to be offered to patients on the NHS in England. About 50 people a year with the inherited blood disorder are likely ...
A little over a year ago, the FDA approved Casgevy, a cellular therapy, for the treatment of sickle cell disease and beta-thalassemia. ... As a treatment administered only once, it carries an ...
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [32] It was approved for medical use in the European Union in May 2019, [33] and in the United States in August 2022.
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.
Casgevy is approved for the treatment of two genetic disorders - sickle cell disease and transfusion-dependent beta-thalassemia - in the United States. The treatment involves patients receiving ...
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