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The U.S. Food and Drug Administration (FDA) granted approval for luspatercept–aamt in November 2019, for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
Luspatercept is a drug used to treat anemia in adults with β-thalassemia, it can improve the maturation of red blood cells and reduce the need for frequent blood transfusions. It is administered by injection every three weeks. Luspatercept was authorised for use in the US in 2019 and by the European Medicines Agency in 2020. [78]
For thalassemia, regular lifelong blood transfusions is the usual treatment. Bone marrow transplants can be curative for some children. [38] Medications like deferoxamine, deferiprone and luspatercept. [39] Gene therapy, exagamglogene autotemcel is approved for medical use in the United Kingdom since November 2023. [40] [41]
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The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.
Counseling is indicated in all persons with genetic disorders, especially when the family is at risk of a severe form of disease that may be prevented. [4] Severe thalassemia : Patients with severe thalassemia require medical treatment. A blood transfusion regimen was the first measure effective in prolonging life. [2]
The regulator, in its so-called complete response letter, said the drug patisiran did not meaningfully improve the condition of patients with heart muscle issues or cardiomyopathy caused by ATTR ...
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.
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