Patient story Experimental Gene Therapy for Beta Thalassemia: Aliya’s Story For the first 30 years of her life, Aliya needed monthly blood transfusions for her inherited blood disorder. A gene therapy changed everything.
Patient story Beta Thalassemia Major: Mia’s Story From the time she was just a few months old, Mia needed blood transfusions due to her rare blood disease. A bone marrow transplant changed everything.
