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.

A very rare condition makes Joyce vulnerable to infections and long-term complications, but she remains wonderfully independent and adventurous.

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

Emma’s bone marrow wasn’t producing what her body needed to fight infections. A bone marrow transplant transformed her life.

Selene is healthy after receiving a bone marrow transplant at CHOP to correct a rare blood disorder that left her unable to fight off bacterial infections.