06-12-2025 13:00 via medpagetoday.com

Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders

(MedPage Today) -- ORLANDO--The gene therapy exagamglogene autotemcel (exa-cel; Casgevy)--already approved for patients 12 years and older with transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD)--led to durable...
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