A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration. Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018. “He’s doing amazing. Boys his age, typically, with Duchenne are on their way to being wheelchair bound,” said Kathryn Edison, Connor’s mother. Connor’s parents first noticed he was struggling with movement in preschool. “He had a hard time getting up off the ground. He was not keeping up with his peers on the playground. We
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