FDA considers new muscular dystrophy treatment using experimental gene therapy : Shots

Susan and Chris Finazzo have enrolled their sons Dylan and Chase in a study of gene therapy for Duchenne muscular dystrophy. The experimental treatment is still being studied but researchers hope it may help prevent the devastating effects of the disease.

Natalia de la Rosa Reyes/Susan Finazzo

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 orthodics 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.

The disease, which almost exclusively affects boys, destroys muscles. Most boys end up in wheelchairs before they become teenagers. Eventually, their hearts and lungs give out. Most people with the disease die in their 30s or 40s. It’s incurable.

“It’s absolutely devastating,” says Susan Finazzo. “You end up going through a mourning process. You’re mourning the loss of the life that your child should have had.”

Finazzo couldn’t help but wonder of her children: “Are they going to be able to go to college? Are they going…