~~<i id='44B53DA659'><strike id='44B53DA659'><tt id='44B53DA659'><pre id='44B53DA659'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

focus

Wikipedia

author：explore 2025-09-30 00:03:30 - browse：85

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： Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies
Next article： WuXi Biologics beefs up its lobbying operation

leisure time

explore

entertainment

Wikipedia

focus

hotspot

AdobeU.S.regulatorsonWednesdayapprovedthefirsttreatmentforanultra-rarediseasethatcausespeopletogrowb

WSS

Clinical trials must include more disabled people

STAT series 'Denied by AI' wins major journalism prizes

Patient advocates sound warning on FDA proposal on salt intake

Cherokee Nation medical school graduates its first class

Eye stem cell transplant could help restore vision in injured people

The pain doctors face in trying to build their families

focus

Wikipedia

leisure time

23andMe had bad news about my health. I wish a person had delivered it

Readout Newsletter: Raw milk, Wegovy tests, Eisai and Biogen

Walensky prepares to leave CDC — and Congress — behind

STAT reporters are Pulitzer Prize finalists for investigative reporting

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

Living with Hashimoto's and celiac disease, and med school, too

Medicare proposal to cover medical devices draws a mixed reaction

HHS suspends federal funding for EcoHealth Alliance

At least 13 dead in Texas as scorching temps continue

Hilary expected to bring catastrophic, life

explore

Ukraine taking heavy casualties 10 weeks into its counteroffensive

What a koala virus tells us about the human genome

Obesity drug from Roche shows promise in early study

Ripples from Flovent's discontinuation is killing children with asthma

Fertility clinic egg and embryo storage is a disaster

STAT series 'Denied by AI' wins major journalism prizes

entertainment

Wikipedia

focus

hotspot