focus -WSS

focus

author：Wikipedia 2025-10-02 20:59:46 - browse：247

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： Cancer drug shortages should be causing more outrage
Next article： Mindstrong's demise and the future of mental health care

WSS

Health tech startups drop sharply from 2021 highs

How one patient went off dialysis — and others can too

Covid vaccines: Private market messier than government rollout

Kaiser Permanente unions announce official strike date

Study finds gene variants tied to breast cancer risk in Black women

As a primary care physician, here's how I hope to partner with AI

explore

focus

entertainment

How one medical school became remarkably diverse

Readout Newsletter: Eli Lilly, AstraZeneca, Bristol Meyers Squibb

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

Digital health: a case of mistaken identity

How to save PrEP access — and even expand it

ASCO eve: Let’s set the table for this year's meeting

Duchenne breakthrough therapy leaves behind pioneering families

H5N1 bird flu outbreak: Second human case reported, in Michigan

Over 800 arrested across France in 3rd night of riots after fatal police shooting of teen

Listen: Who gets the credit for GLP

hotspot

Digital health: a case of mistaken identity

Mindstrong's demise and the future of mental health care

Eyeing GLP

H5N1 virus can be tracked in retail milk, scientists say

Paxlovid should be available without a positive Covid test

As a primary care physician, here's how I hope to partner with AI

explore

leisure time

Wikipedia

entertainment