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  2. Management of thalassemia - Wikipedia

    en.wikipedia.org/wiki/Management_of_thalassemia

    This study showed that long-term treatment did result in a sustainable reduction in the iron burden faced by patients receiving blood transfusions for thalassemia. [24] An additional benefit of the use of deferasirox instead of desferoxamine is that, unlike desferoxamine, early studies have indicated that deferasirox does not have a significant ...

  3. Betibeglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Betibeglogene_autotemcel

    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.

  4. Exagamglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Exagamglogene_autotemcel

    The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [9] [10] [11] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [12 ...

  5. FDA clears first CRISPR treatment for a second disease, beta ...

    www.aol.com/fda-clears-first-crispr-treatment...

    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.

  6. Thalassemia - Wikipedia

    en.wikipedia.org/wiki/Thalassemia

    β thalassemia major (Mediterranean anemia or Cooley anemia) is caused by a β o /β o genotype. No functional β chains are produced, and thus no hemoglobin A can be assembled. This is the most severe form of β-thalassemia; β thalassemia intermedia is caused by a β + /β o or β + /β + genotype. In this form, some hemoglobin A is produced;

  7. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Beremagene geperpavec (Vyjuvek): treatment of wounds. [2] 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 ...

  8. List of therapeutic monoclonal antibodies - Wikipedia

    en.wikipedia.org/wiki/List_of_therapeutic...

    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.

  9. Luspatercept - Wikipedia

    en.wikipedia.org/wiki/Luspatercept

    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]

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