STAT+: With an age cutoff for a new gene therapy, families and doctors scramble to treat kids before their 6th birthday
To families whose boys just turned 6, putting them over the line for Sarepta's gene therapy for muscular dystrophy felt like the "Hunger Games," as one parent put it.
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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