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：
Living with Hashimoto's and celiac disease, and med school, too
Next article： Sports medicine is finally prioritizing gender equality
Next article： Sports medicine is finally prioritizing gender equality
