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          hotspot

          hotspot

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          Gene therapy helped Conner Curran, right, walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. -- Biotech coverage from STAT
          Gene therapy helped Conner Curran (at right) walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. Courtesy Jess & Chris Curran

          In the beginning, Conner Curran ran up the stairs.

          It was a miraculous moment for him and his parents, Jess and Chris Curran. Just months before, contractors were in their New York home to install a motorized lift, while Jess tearfully explained that it wasn’t for a grandparent but for their 7-year-old son, who suffered from Duchenne muscular dystrophy and could barely haul himself up the steps.

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          Then Conner was accepted as the first patient in Pfizer’s Duchenne gene therapy trial. Three weeks later, he could bound to the second floor. Soon, he could walk the two-plus miles to Goldberg’s, a local bagel shop. “I couldn’t do that before,” he told NPR in 2020. 

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