~~<i id='B04C9089F9'><strike id='B04C9089F9'><tt id='B04C9089F9'><pre id='B04C9089F9'></pre></tt></strike></i>~~

Home» focus» comprehensive

comprehensive

author：Wikipedia 2025-09-29 09:42:30 Page View：9913

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： Novartis develops a rapidly manufactured CAR
Next article： Bright Health sells Medicare Advantage business to Molina

explore

Medicare proposal to cover medical devices draws a mixed reaction

AdobeThemedicaldeviceindustryissplitonwhetheraproposednewMedicarereimbursementpathwaywilldoanythingt

Charges dismissed against white woman who spat on Black woman during protests in Connecticut

HARTFORD,Conn.--HARTFORD,Conn.(AP)—AjudgeFridaydismissedhatecrimeandotherchargesagainstawhitewomanwh

Cancer drug shortages should be causing more outrage

DrugshortagesareagrowingproblemintheU.S.,andashortageoflivesavingcancerdrugsinparticularhasreachedcr

FDA gives detailed accounting of AI

AdobeTheFoodandDrugAdministrationonThursdayreleasedanewaccountingofartificialintelligencetoolscleare