entertainment -WSS

entertainment

author：Wikipedia 2025-09-27 07:46:22 - browse：7528

Sarepta - Cambridge -AP — A logo sign outside of the headquarters of Sarepta Therapeutics in Cambridge, Mass. Kristoffer Tripplaar/Sipa via AP

Reviewers at the Food and Drug Administration were leaning toward rejecting a closely watched gene therapy for Duchenne muscular dystrophy made by Sarepta Therapeutics, prompting a top official to intervene earlier this year, according to three people with direct knowledge of the agency deliberations.

Inside the FDA office that oversees gene therapies, some staff had reached a non-binding conclusion that Sarepta’s gene therapy should be rejected, the individuals said, speaking on condition of anonymity. Peter Marks, a top FDA official and vocal advocate for faster gene therapy approvals, stepped in and directed staff to schedule a public hearing on the therapy on May 12.

Sarepta announced the scheduling of the public hearing last month, but the internal deliberations that led to it have not been previously reported.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Duchenne breakthrough therapy leaves behind pioneering families
Next article： WATCH: Why do fingernails fall off when they're damaged?

hotspot

WSS

U.K. considers world's first use of meningitis vaccine to address gonorrhea

With FDA staff opposed to Sarepta therapy, top official intervened

Readout Newsletter: Bayer study halts, Carmot to IPO, and more

Readout Newsletter: Bayer study halts, Carmot to IPO, and more

PBM reforms clear House panel

#NeedHerScience: shining a light on gender disparities in medicine

focus

entertainment

knowledge

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Anthos stroke drug appears safer than common blood thinner

Drug repurposing or repositioning? The language matters

#NeedHerScience: shining a light on gender disparities in medicine

How one medical school became remarkably diverse

PBM reforms clear House panel

How one medical school became remarkably diverse

Readout LOUD podcast: A CRISPR milestone, biotech mixology

ChatGPT in medicine: STAT answers readers' burning questions

Donald Trump's relentless attacks on special counsel Jack Smith

Wikipedia

Gain of function research would be limited under House proposal

Why medical students are struggling with mental health

FDA grants conditional OK to Sarepta’s gene therapy for Duchenne

Readout Newsletter: Bayer study halts, Carmot to IPO, and more

In areas with more Black doctors, Black people live longer

Morphosys mixed study results for blood cancer drug

hotspot

Wikipedia

focus

Wikipedia