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.

A seemingly benign heart murmur after birth prompts a newborn’s parents to pursue diagnosis and treatment at Children’s Hospital of Philadelphia (CHOP).

An FDA-approved gene therapy eliminates acute pain episodes for people with sickle cell disease and transforms their life expectancy.

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.