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.


Gene Therapy for Duchenne Muscular Dystrophy Cash’s Story

Jena: This is Cash. He's six years old. He's a very happy little boy. Cash has Duchenne Muscular Dystrophy. He was diagnosed just before his second birthday.

Susan Matesanz, MD: Cash is getting a treatment called Alevitis or D Land Dystrogene Moxiparbevac. It's a microdystrophin gene therapy. And the hope is that by giving this medication, his body will start to make a modified version of the protein that he's not currently making.

Phillip: This is a blessing. It's a dream come true. We've been watching this come to be since he was two years old. We had so much support through CHOP. The doctors actually advocated for us.

Susan Matesanz, MD: This is the first time we have a treatment to offer many patients that will hopefully slow the progression of this disease.

And so our hope is that with this treatment, boys will walk longer, that there'll be delayed onset of heart and lung problems, and that we'll hopefully then be able to have future treatments to change the natural trajectory of what this disease looks like.

Jena: For the first time in a long time, we're going to be able to enjoy him as a little boy and watch him grow and hopefully run and keep up with his brother and his older sister.

And we're really just kind of looking forward to him having physical freedom.

Provider: Can I give you a hug?

Jena: It just feels like what we were so fearful of is now just replaced with a feeling of hope.

Topics Covered: Muscular Dystrophy

Related Centers and Programs: Gene Therapy for Neurological Disorders , Neuromuscular Program, Neuroscience Center, Clinical In Vivo Gene Therapy