Division of Hematology Patient Stories

An FDA-approved gene therapy is giving people with sickle cell disease a future they didn’t think they’d have.

Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.

For 50-plus years, Jay dealt with the frequent treatments needed for his hemophilia. A gene therapy put an end to the treatments and to the worry the disease caused him.

For nearly 50 years, Bill needed frequent treatment for hemophilia — often every few days. A gene therapy changed everything.

Born with a rare condition affecting her bone marrow, 2-year-old Elizabeth has large care teams at CHOP and her local hospital who coordinate to keep her growing and happy.

For years, sickle cell disease caused frequent pain crises and hospitalizations for Asher. A curative therapy has eliminated those harrowing experiences.

Sickle cell disease brings constant fear of life-threatening complications. A successful bone marrow transplant ended those fears for Dallas and her family.

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.

For years, Ella’s health seemed fragile, culminating in a life-threatening infection. She now needs injections to control her rare blood disorder.