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.
advertisement
“Oh, crap,” Secrist said to herself.
Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!
GET STARTED Log In
Previous article：
Alzheimer's risk factor APOE4 may be a distinct form of the disease
Next article： Moonlake's readout produced a cash windfall. Risks remain
Next article： Moonlake's readout produced a cash windfall. Risks remain
