Clinical In Vivo Gene Therapy Resources

Caring for a child with any illness or injury can be overwhelming. To help you find answers to your questions and feel confident with the care you’re providing your child, we’ve created the following list of health resources. We hope they can help make this time in your family’s life a bit easier.

Videos

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

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.