Disease or Syndrome: Anemia, Cooley

Medical news and research articles

Gene therapy leads to improved quality of life in patients with sickle cell disease and beta thalassemia

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two ...

Aug 27, 2025

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FDA approves exagamglogene autotemcel to treat beta-thalassemia

After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat patients older than 12 years ...

Jan 17, 2024

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