With these therapies, the stem cells are taken from the patients themselves, and then the stem cells are edited or modified in a way that offers clinical benefit for the patient... having more fetal hemoglobin reduces complications of thalassemia and sickle cell disease.
More than 90 percent of patients with beta thalassemia treated with exa-cel were able to stop the blood transfusions they were dependent on before the treatment, according to the study.
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