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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 .
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]
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage. See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page.
Recently, the U.S. Food and Drug Administration (FDA) granted the Regenerative Medicine Advanced Therapy (RMAT) designation to the company's CTX112, a potential therapy for certain B-cell ...
Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia [3] Brexucabtagene autoleucel (Tecartus): treatment for mantle cell lymphoma and acute lymphoblastic leukemia [4] [5] Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor peripheral artery disease
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 gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
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