~~<i id='3C346B8339'><strike id='3C346B8339'><tt id='3C346B8339'><pre id='3C346B8339'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

explore

fashion

author：knowledge 2025-09-29 22:37:14 - browse：57825

Sarepta Therapeutics said Monday afternoon that its gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial — likely a major disappointment for patients and doctors who have been desperately awaiting the treatment for years.

The company said all patients in the study improved and that secondary measurements indicated the drug was having an effect. Nevertheless, the results are likely to raise questions about whether the Food and Drug Administration will expand access to the medicine.

Trading in Sarepta shares was halted at $107 with the release of the study results.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Continuity nursing helped my family in our darkest PICU moments
Next article： Cue Health, Covid

explore

Wikipedia

knowledge

hotspot

Wikipedia

ThisistheonlineeditionofAdam’sBiotechScorecard,asubscriber-onlynewsletter.STAT+subscriberscansignuph

WSS

Liver decline slowed by off

Reports of telehealth's death have been greatly exaggerated

Investigation reveals sickle cell patients pressured into sterilizations

Review: 'Barbie' will hit you like a shot in the heart

As a primary care physician, here's how I hope to partner with AI

Caitlin Bernard's story shows physicians can't shy from advocacy

explore

fashion

explore

How one medical school became remarkably diverse

Cue Health, Covid

New air purifier tech could be key to improving indoor air quality

A double vaccine crisis is endangering millions of children

After affirmative action ruling, medical educators look to 'holistic review'

Fentanyl is making it harder to start addiction treatment

Cancer drug shortages should be causing more outrage

Why an extreme heat and wildfire smoke combination can be so dangerous

New air purifier tech could be key to improving indoor air quality

Why an extreme heat and wildfire smoke combination can be so dangerous

Wikipedia

Liver decline slowed by off

Questions and answers about ramping up H5N1 bird flu vaccine

Our daughter's deadly disease: trisomy 18. We wanted to protect her

Scrutiny of post

Bird flu surveillance in wastewater gets boost from consortium

Addressing health care workers' trauma can help fight burnout

knowledge

hotspot

hotspot

Wikipedia