focus -WSS

focus

author：explore 2025-09-03 11:45:16 - browse：1348

Sarepta - Cambridge -AP — Kristoffer Tripplaar/Sipa via AP

The Food and Drug Administration granted conditional approval Thursday to the first gene therapy for Duchenne muscular dystrophy. Regulators restricted the treatment to younger patients, with additional data required to broaden its use.

The gene therapy, called Elevydis, is made by Sarepta Therapeutics. The company will charge $3.2 million for the treatment, making it the U.S.’s second most expensive drug, behind a recently approved gene therapy for hemophilia. CEO Doug Ingram said on a conference call the price was below what a recently published company-funded analysis found would be cost-effective.

“The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevydis is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” Ingram said.

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

GET STARTED Log In

Previous article： State Dept. review finds Biden bears some blame for Afghanistan failures
Next article： Adam's Take: Intra

hotspot

WSS

Chinese researcher claims birth of first gene

Medical devices makers are asking for forgiveness, not permission

The EPA's new lead rule: How one expert crunched the numbers

As biotech industry slumps, bioscience job market is upside down

Yancopoulos of Regeneron works to expand Dupixent success

Chinese researcher claims birth of first gene

focus

focus

focus

Walensky prepares to leave CDC — and Congress — behind

Asbestos ban is immediate, but health effects will linger for decades

Moonlake's readout produced a cash windfall. Risks remain

With clinical trial data readouts, here's how to spot spin

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

What to do when lifesaving products contribute to climate change

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

Yancopoulos of Regeneron works to expand Dupixent success

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

As biotech industry slumps, bioscience job market is upside down

hotspot

Testing GLP

Bring on the sabbaticals for nurses

FDA investigates CAR

How to pay for weight loss drugs and save Medicare

Diagnostic tests are hitting their mathematical limits

Fetal surgery at risk from Dobbs and new abortion restrictions

hotspot

Wikipedia

hotspot

hotspot