
WASHINGTON (AP) — The Food and Drug Administration said Friday it will add a new warning and other limitations to a gene therapy for Duchenne’s muscular dystrophy that's been linked to two patient deaths.
The infused therapy from Sarepta Therapeutics will carry a boxed warning — the most serious type — alerting doctors and patients to the risk of potentially fatal liver failure with the treatment, the FDA said in a release.
The one-time therapy, Elevidys, has been under FDA scrutiny since the company reported the first of two deaths of teenage boys in March. Following a second death reported in June, the FDA briefly called for halting all shipments of the drug. But the agency quickly reversed course after facing pushback from patient families and libertarian activists close to President Donald Trump.
Elevidys is the first U.S.-approved gene therapy for Duchenne’s muscular dystrophy, a fatal muscle-wasting disease that affects boys and young men.
In addition to the boxed warning, the FDA is also limiting the drug's approved use to patients who are 4 years old and up and can still walk. Previously the FDA had allowed the drug's use in immobile patients, who generally have more advanced disease.
New labeling will also recommend weekly liver function monitoring for the first three months of treatment, as well as other precautionary steps.
Elevidys is Sarepta's best-selling product and recent headwinds against the drug have weighed heavily on the company and its stock. In July, the Cambridge, Massachusetts-based company announced it would lay off 500 employees.
Sarepta Therapeutics Inc. shares rose 7.7% in trading after the FDA announcement, reflecting improved visibility for investors about the company's outlook.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
NEUESTE BEITRÄGE
- 1
6 Pet Sitting Administrations for Your Cherished Pets06.06.2024 - 2
Mexico says a third of 130,000 missing people might be alive, fueling criticisms by families27.03.2026 - 3
Iran War Derails The Automotive Industry27.03.2026 - 4
Famous Restroom Beautifying Styles For 202405.06.2024 - 5
Rubble, mud and hair: How to rebuild a home in Gaza03.04.2026 - 6
Between 600 to 800 aid trucks entering Gaza daily since start of ceasefire, COGAT confirms17.12.2025 - 7
NASA releases new ‘Earthset’ and eclipse images taken during historic flyby of the moon07.04.2026 - 8
Best Streaming Gadget for Your Home Theater01.01.1 - 9
6 Modest and Strong Tire Brands05.06.2024 - 10
Middle East hotels hit pandemic-era lows amid Iran war07.04.2026 - 11
NASA releases stunning first images of Earth taken by the Artemis II astronauts03.04.2026 - 12
Eurasian cargo hub expands to capture Europe-Asia freight flows03.04.2026 - 13
Horses really can smell our fear, new study finds14.01.2026 - 14
Figure out How to Adjust Work, Life, and an Internet based Degree17.10.2023 - 15
American tourists left stranded in the Caribbean following flight cancellations after airspace closed for Maduro operation05.01.2026
Ähnliche Artikel
Artemis 2 captures historic 'Earthset' photo | Space photo of the day for April 7, 202607.04.2026
Tesla Germany Registrations Quadruple to 9,252 Vehicles in Best March Ever07.04.2026
What's going on with Katseye? The Manon Bannerman hiatus drama, explained.07.04.2026
Two IDF officers, civilian face indictment in alleged Gaza aid-truck smuggling scheme07.04.2026
West Palm Beach Shorecrest, renderings of downtown waterfront condo07.04.2026
Artemis II astronauts make long-distance call to the space station as they head home from the moon07.04.2026
Tuesday, April 7. Russia’s War On Ukraine: News And Information From Ukraine07.04.2026
The Eastern Bongo, Kenya’s Rare Forest Antelope on the Brink07.04.2026
U.K. blocks Kanye West from entering Britain to headline now canceled festival: What led to the ban07.04.2026
Russia confirms 16 Cameroonian soldiers killed in Ukraine war07.04.2026
First Phosphate advances battery-grade phosphate project as analysts highlight strategic Federal support07.04.2026
After fleeing past Hezbollah fighting, some Israelis on northern border vow to stay07.04.2026
'Unreal' solar eclipse: Artemis 2 crew just saw one of the rarest sights in spaceflight history07.04.2026
Chinese construction workers in Israel: 'I’d rather be bombed than live in poverty'07.04.2026
Broken toilet, T-shirts on windows and collecting saliva: The weirdness of daily life aboard Orion07.04.2026
Czech Republic caps fuel prices amid Iran war energy crisis07.04.2026
NASA releases stunning new images captured by the Artemis II moon mission, including 'Earthset' and a solar eclipse from space07.04.2026
Amid Iran war, 53 of Israel's future scientists showcase projects in Jerusalem contest07.04.2026
Iranian-linked drone attack kills Kurdish couple in northern Iraq07.04.2026
Thousands of small fish defy gravity to climb Congo waterfall07.04.2026


































