~~<i id='74AF8CCD47'><strike id='74AF8CCD47'><tt id='74AF8CCD47'><pre id='74AF8CCD47'></pre></tt></strike></i>~~

Home» comprehensive» focus

focus

author：explore 2025-10-01 15:44:10 Page View：698

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： HIPAA doesn't protect reproductive records from prosecutors
Next article： How to save PrEP access — and even expand it

explore

In age of alternative facts, a scholarly course on calling out crap

Screenshotviacallingbullshit.orgTiredofalternativefacts,fakenews,andbreathlesshyperbole,twoprofessor

Death toll from an explosion and collapse of a Paris building in June climbs to 3

PARIS--Awomaninher70swhoworkedatadesignschoolhasdiedastheresultofanexplosionincentralParisamonthagot

In age of alternative facts, a scholarly course on calling out crap

Screenshotviacallingbullshit.orgTiredofalternativefacts,fakenews,andbreathlesshyperbole,twoprofessor

Senate HELP committee spars over health care workforce policies

Sen.BillCassidy(R-La.),left,andSen.BernieSanders(I-Vt.),right,clashedoverabilltoincreaseranksinthehe

WSS

focus

focus

leisure time

explore

Senate HELP committee spars over health care workforce policies