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Exa-cel gene therapy may off effective cure for beta-thalassemia and sickle cell disease in children younger than 12

[ Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12. Researchers say the therapy’s potential to offer a cure at an early age—before organ damage accumulates—could make exa-cel even …

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