Ann and Robert H. Lurie Children's Hospital: Gene Therapy for Thalassemia Ends Need for Transfusions in Young Children
February 23, 2022
February 23, 2022
CHICAGO, Illinois, Feb. 23 (TNSJou) -- The Ann and Robert H. Lurie Children's Hospital issued the following news release:
Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in age 4-34 ye . . .
Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in age 4-34 ye . . .