Safety and efficacy assessments clinical trials

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

This study is looking at the long-term effects of a gene therapy treatment for people with a condition called beta-thalassemia. It follows patients who have already received the treatment in earlier studies to see how they are doing over many years, checking their safety and how well the treatment continues to work.