Facing a devastating disease, Adam’s family enrolled him in a gene therapy clinical trial at CHOP. Despite complications, the therapy shows the promise of precision care for rare diseases.

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.

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

Khymir is a 4-year-old boy who was found to have adrenoleukodystrophy (ALD) through Pennsylvania’s newborn screening.

Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.

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.

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