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In mid-July, Kristen Secrist hopped on a call with her 5-year-old son’s doctor, who had urgent news: The first gene therapy had been approved for Duchenne muscular dystrophy. Her son, Hiram, would be a perfect candidate — if, she added, they could get him dosed in time.

The treatment was approved only for 4- and 5-year-olds. Hiram turned 6 in three weeks. 


“Oh, crap,” Secrist said to herself.

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