Gene therapy for muscular dystrophy stirs hopes and controversy

https://www.npr.org/sections/health-shots/2023/05/02/ [login to see] /gene-therapy-for-muscular-dystrophy-stirs-hopes-and-controversy
When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. But they didn't think it was anything serious.

"He would fall a lot. Not like a lot a lot. But he had trouble climbing playground equipment. He fell down the stairs at his pre school a couple of times," says his mother, Susan Finazzo, 40, who lives in Miami.

Chase's younger brother, Dylan, was more agile. But he started walking a little late.

"We're not thinking anything of it. He's just got some weak ankles. He's got some little tiny little orthotics for toddlers," she says. "He's sees an orthopedic surgeon. We've got PT. We're all over it."

So Susan and her husband Chris Finazzo were stunned when doctors told them both boys had Duchenne muscular dystrophy. While rare, the disease is the most common inherited neuromuscular disorder among children. It affects an estimated 10,000 to 12,000 children in the U.S.