4-year-old SoCal boy sees stunning improvement after undergoing gene therapy for Duchenne muscular dystrophy
The game-changing treatment had a huge impact on one local family.
"He was behind but according to the pediatrician, because he's a boy, they're slower," said Zac Kargar, Thomas' father. "With time, the patients will eventually stop walking. It will affect their heart, it will affect their ability to breathe, to eat, and eventually they die prematurely," said Dr. Leigh Maria Ramos-Platt, program director for
"Because there's a defective gene, we give the patients a replacement gene. We infect them with a better gene to produce a protein that they are missing to hopefully improve their strength, keep them walking and help them stay healthier," said Ramos-Platt."He jumped for the first time and he's never been able to jump in his life," said Orellana.
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