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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Institute of Hematology & Blood Diseases Hospital, China · 2023
ClinicalTrials.gov · Datasets · License: Public Domain · 2023
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cohort-studygene-therapy
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.
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