Gene Therapy for Beta Thalassemia: Rahemeen’s Story
Rahemeen no longer needs regular blood transfusions, and her future has been transformed, ever since she received a curative gene therapy for her inherited blood disorder.
Thalassemia Center Patient Stories
Every person on your child's team has the same goal: to see your child thrive. We provide medical care, emotional counseling and much more. We make sure your entire family gets the support you need.
Appointments and referrals
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.
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.
Beta Thalassemia Major: Nicholas's Story
Beta thalassemia major forced Nicholas to undergo regular blood transfusions until he received a lifesaving bone marrow transplant.