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

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

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.

18-month-old Josie received a stem cell transplant to stop a rare and devastating genetic condition in its tracks.

Diagnosed before his first birthday with a rare disease that causes ongoing damage to cells and organs, Ethan quickly received the only treatment that prevents further disease.

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.

Treatment for childhood leukemia has had several long-term effects on Raine’s health, but this successful college graduate is pursuing her dream.

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

"CHOP is so great because they support the whole family," says Gabby's dad, Paul.