focus -WSS

focus

author：comprehensive 2025-09-30 12:11:15 - browse：41

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： FDA approval of Sarepta Duchenne genetic therapy gives me hope
Next article： With clinical trial data readouts, here's how to spot spin

WSS

What Mike Pence has said that could be used in a Donald Trump trial

ReNAgade cuts staff seven months after raising $300 million

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

A cyberattack has disrupted hospitals and health care in several states

Readout Newsletter: The latest in Pfizer, Moderna, and Sanofi

Rep. Raul Ruiz on the physician shortage and more

focus

focus

focus

Bright Health sells Medicare Advantage business to Molina

What the cancer research community can learn from the Covid response

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

What don’t we know about an Alzheimer’s drug side effect?

What does generative AI mean for health care? We asked experts

U.K. Biobank has 500,000 peoples' genome sequences available

Affirmative action in medical school literally saved lives

Trump drops motion seeking removal of DA probing efforts to overturn 2020 election in Georgia

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

Pediatric cancer treatments threatened by a new FDA policy

hotspot

Rep. Raul Ruiz on the physician shortage and more

Readout Newsletter: The latest in Pfizer, Moderna, and Sanofi

A cyberattack has disrupted hospitals and health care in several states

What Henrietta Lacks settlement says about racism in medicine

Saudi VC loops Novo Nordisk, biotech VCs into longevity incubator

OTC birth control: A brief history of over the counter pill design

hotspot

leisure time

focus

focus