~~<i id='B66CD643F1'><strike id='B66CD643F1'><tt id='B66CD643F1'><pre id='B66CD643F1'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

explore

knowledge

author：leisure time 2025-09-29 19:58:22 - browse：68

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： Bright Health sells Medicare Advantage business to Molina
Next article： A new approach to psychiatric drug development

knowledge

focus

leisure time

knowledge

focus

MarioTama/GettyImagesLONDON—NovoNordisk’sdiabetesmedicationOzempiccuttheriskofpatientsdevelopingadva

WSS

Congress: Eradicate hepatitis C and reduce the deficit

Immune cells and nerve overgrowth may explain recurring UTI pain

$1 billion proposal to grow Boston, Massachusetts biotech

Change Healthcare cyberattack affects 25% of CVS/Aetna insurance claims

Who gets to use the title 'doctor'?

Congress: Eradicate hepatitis C and reduce the deficit

explore

knowledge

knowledge

Novavax promises a turnaround & Lilly roils the obesity market

Medicare drug price negotiation continues, as pharma counteroffers

Trump demands the U.S. pay no more for drugs than other countries … again

Grail faces three suits alleging harassment, retaliation

Sports medicine is finally prioritizing gender equality

Immune cells and nerve overgrowth may explain recurring UTI pain

Guidelines to prevent youth baseball injuries need more muscle

Congress likely to miss an opportunity to fix drug shortages issue

Apple is now the first public company to be valued at $3 trillion

A new approach to psychiatric drug development

focus

For AI in health care standards, federal regulators join with industry group

Special counsel informs Trump he is target in probe of efforts to overturn 2020 election

Families look for replacement for GSK asthma drug Flovent

A new approach to psychiatric drug development

Longer MASH drug regimen improved liver, Akero study finds

Why police arrested suspected Gilgo Beach killer away from his house

focus

leisure time

knowledge

focus