![]() ![]() ![]() ![]() So if this moves the needle in their life expectancy - even if it does it for a day - it's worth it."īut there's an intense debate about this. I want them to be alive long enough to enjoy what life is. You don't care about the not walking," Finazzo says. At the end of the day as a Duchenne parent, you don't even care about the wheelchair anymore. "I hope that this can extend their lives a little bit. The hope is this "micro-dystrophin" will at least help slow the progression of the disease. The gene produces a miniature version of a protein called dystrophin, that boys with muscular dystrophy are missing or don't have enough of. The gene therapy works by infusing trillions of harmless viruses that has been genetically modified to ferry a gene to patients' muscles. Shots - Health News Drugmakers are slow to prove medicines that got a fast track to market really work Most people with the disease die in their 30s or 40s. Eventually, their hearts and lungs give out. Most boys end up in wheelchairs before they become teenagers. The disease, which almost exclusively affects boys, destroys muscles. It affects an estimated 10,000 to 12,000 children in the U.S. While rare, the disease is the most common inherited neuromuscular disorder among children. So Susan and her husband Chris Finazzo were stunned when doctors told them both boys had Duchenne muscular dystrophy. He's got some little tiny little orthotics for toddlers," she says. He fell down the stairs at his pre school a couple of times," says his mother, Susan Finazzo, 40, who lives in Miami.Ĭhase's younger brother, Dylan, was more agile. But he had trouble climbing playground equipment. But they didn't think it was anything serious. When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. The experimental treatment is still being studied but researchers hope it may help prevent the devastating effects of the disease. Susan and Chris Finazzo have enrolled their sons Dylan and Chase in a study of gene therapy for Duchenne muscular dystrophy. ![]()
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