Patient stories

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.

For the first 30 years of her life, Aliya needed monthly blood transfusions for her inherited blood disorder. A gene therapy changed everything.

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 forced Nicholas to undergo regular blood transfusions until he received a lifesaving bone marrow transplant.