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Patients with beta thalassemia minor are usually asymptomatic and are often monitored without treatment. [8] Beta thalassemia minor may coexist with other conditions such as chronic hepatitis B, chronic hepatitis C, non-alcoholic fatty liver disease and alcoholic liver disease that, when combined or co-existing, may cause a person to have iron ...
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [33] It was approved for medical use in the European Union in May 2019, [34] and in the United States in August 2022.
β thalassemia minor is caused by a β/ ... Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [58]
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.
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]
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. [5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [8]
Minor slumps like these have been rare for the company in the past few years. ... sickle cell disease and transfusion-dependent beta thalassemia. ... Povetacicept is in phase 3 testing as a ...
Beta-thalassemia is a genetic disease mostly caused by beta-globin gene mutations. [18] Clinical diagnosis is based on interpretation of the peripheral blood smear, which examines red blood cell morphology, followed by hemoglobin analysis and confirmed by DNA sequencing.
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