fashion -WSS

fashion

author：leisure time 2025-09-30 00:51:07 - browse：56481

FDA's Peter Marks talks with STAT's Adam Feuerstein — Peter Marks, the FDA's top regulator of gene therapies (right), speaks with STAT's Adam Feuerstein in San Francisco. Sarah Gonzalez/Peopletography for STAT

SAN FRANCISCO — Peter Marks, the Food and Drug Administration’s top regulator of gene therapies, seems very comfortable with granting some form of full approval to Elevidys, the Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy, based on his comments to me on Monday night during our “fireside chat” at a STAT event.

To be clear, Marks declined to comment specifically about the ongoing FDA review of Elevidys. I tried. However, Marks described a “hypothetical case” in the context of my Sarepta questions that clearly suggested the failure of an Elevidys confirmatory study in October didn’t bother him all that much.

“Hypothetically, if you had a product that used an aggregate scale and all of the components of that aggregate scale looked pretty good, or nearly all of them look pretty good, but the aggregate, for various reasons, didn’t come out perfectly. We approved products based on that,” Marks told me.

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

Subscribe Log In

Previous article： Malaria cases in Florida and Texas: Here’s what you need to know
Next article： Alabama frozen embryo ruling has major implications for IVF clinics

WSS

NIH study of ME/CFS points to clear biological hallmarks

Medicare inequities exacerbate Puerto Rico’s health disparities

Medicare inequities exacerbate Puerto Rico’s health disparities

Readout Newsletter: Wegovy, dialysis, Humira, Vir, and more

The Readout: Abbvie and Humira, CRISPR in utero, Annovis Bio

Ocular Therapeutix moves raise hopes, and Alnylam tries to calm some nerves

knowledge

fashion

hotspot

Guidelines to prevent youth baseball injuries need more muscle

He's a Nobel laureate. Critics say he was misleading on Covid

In age of alternative facts, a scholarly course on calling out crap

Ocular Therapeutix moves raise hopes, and Alnylam tries to calm some nerves

Medical leaders decry Supreme Court decision on affirmative action

Ed Silverman on new gene therapies, biosimilars, coffee, and more

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

Alabama hospital puts IVF treatments on hold

Wildfire smoke exposes gaps in outdoor worker protections

Ocular Therapeutix moves raise hopes, and Alnylam tries to calm some nerves

hotspot

Why palliative psychiatry is worth discussing

A new nitrogen execution bill also undermines medical licensing

Ocular Therapeutix moves raise hopes, and Alnylam tries to calm some nerves

Ocular Therapeutix moves raise hopes, and Alnylam tries to calm some nerves

The Readout: Abbvie and Humira, CRISPR in utero, Annovis Bio

Alabama IVF ruling threatens families facing fatal genetic disease

entertainment

Wikipedia

explore

Wikipedia