knowledge -WSS

knowledge

author：fashion 2025-09-29 20:51:46 - browse：134

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： Jerome Adams on the need for new antivirals for Covid
Next article： With fresh data, a dynamic duo powers Eli Lilly to new heights

explore

WSS

Fauci's latest grilling in Congress: 9 hours, behind closed doors

How corporate America is slashing DEI workers amid backlash to diversity programs

Physician assistants need a new name. Here's what it should be

Why Titanic fascinates society, still drawing tours to the wreckage

In shift, Texas to cover initial consult for Afghan baby's gene therapy

Zelenskyy on how Russian invasion may end, gratitude for US aid and Putin's weakness

leisure time

knowledge

focus

Wildfire smoke exposes gaps in outdoor worker protections

FDA early review: GLP

Trump demands the U.S. pay no more for drugs than other countries … again

At JPM 2024, hospital systems pitch financial strength to investors

Anesthesiologist group: stop taking Ozempic before surgery

Kansas must stop changing trans people's sex listing on driver's licenses, judge says

Medical records are filled with copy

Mayo Clinic partners with supercomputer AI firm Cerebras at JPM

Moonlake's readout produced a cash windfall. Risks remain

Sen. Bill Cassidy on how to lower drug prices

hotspot

Why Titanic fascinates society, still drawing tours to the wreckage

Drinking water from half of US faucets contains PFAS: Study

American taxpayers paid $1.8 trillion in health care costs in 2022

Medicare Advantage overpayments discussion gets experts fired up

JPM pitch from hospitals? Revenue streams outside of patient care

JPM pitch from hospitals? Revenue streams outside of patient care

focus

explore

focus

Wikipedia