Cellular Therapy and Transplant Section Patient Stories

After losing two children to a rare genetic disease, an Alaska family finds hope in gene therapy at CHOP, a leader in newborn screening advocacy.

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.

Two hospitals work together to care for a 3-year-old with cancer and his family.

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.

Diagnosed with leukemia at 3 months old, Asa received a revolutionary therapy that was pioneered at CHOP.

Genetic sequencing revealed that Rafi had an extremely rare, often fatal disease — and the discovery was amazingly made before he had serious symptoms.

After multiple rounds of chemotherapy couldn’t wipe out Johnny’s cancer, he came to CHOP and received two versions of CAR T-cell therapy.