~~<i id='C82F3CCA69'><strike id='C82F3CCA69'><tt id='C82F3CCA69'><pre id='C82F3CCA69'></pre></tt></strike></i>~~

Home» focus» knowledge

knowledge

author：hotspot 2025-10-02 06:47:39 Page View：1294

Hiram Secrist (left), who has Duchenne muscular dystrophy, with his mother, Kristen Secrist (center), and his grandmother, Terrie Jordan. Courtesy Secrist family

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.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： At Orexo, a ‘turning point’ for everything but digital health
Next article： The cancer drug shortage isn’t new — and neither are the solutions

focus

How to save PrEP access — and even expand it

UndertheAffordableCareAct,healthinsurersarerequiredtocoverallcostsassociatedwithpreventivecare—inclu

AI can’t replace a physician’s gut feeling

AdobeI’veheard“WebMDsaiditcouldbecancer”countlesstimesinmy15yearsworkingasanemergencymedicinephysici

Walensky prepares to leave CDC — and Congress — behind

OutgoingCDCDirectorRochelleWalenskyJIMWATSON/AFPviaGettyImagesRochelleWalensky,theoutgoingdirectorof

Cosmetics set for new safety regulation. Why the industry is on board

AlbertoPellaschiar/APWASHINGTON—ForthosealreadyfelledbyfumesfromtheBrazilianBlowout,itmightbesmallco