knowledge -WSS

knowledge

author：Wikipedia 2025-09-28 16:35:13 - browse：9

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： How to save PrEP access — and even expand it
Next article： EU regulator recommends expanding Mounjaro approval to obesity

hotspot

WSS

Amylyx ALS drug failure raises questions and concerns

Eli Lilly, Pfizer, and others want in on the online prescription market

The drug industry has found itself all alone in Washington

Amylyx ALS drug failure raises questions and concerns

IVF, abortion rights issues kick off Biden’s State of the Union

UnitedHealth quietly bought dozens of outpatient centers

knowledge

knowledge

Wikipedia

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

Former medical device CEO who sold dummy, plastic parts convicted of fraud

Drug repurposing or repositioning? The language matters

What it’s like to watch children die of measles

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

Think you might have Covid

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Francis Collins on trust in science and how Covid communications failed

Medical leaders decry Supreme Court decision on affirmative action

With psoriasis trial ahead, Alumis raises $259 million

hotspot

What to make of Gilead’s Trodelvy troubles and Elevation Oncology’s rise

NEJM study link tiny plastics in blood vessels to heart problems

Biotech venture capitalists face new demands from investors

AI generated plenty of interest at JPM but not a lot of trust

Francis Collins on trust in science and how Covid communications failed

How to bring more disabled researchers into academia

focus

focus

hotspot

hotspot