
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
Artemis 2 astronauts fly around the moon in record-breaking lunar loop by NASA06.04.2026 - 2
Inside the alleged Russian operation to trigger anti-government protests in Angola26.03.2026 - 3
Europe's powerful Ariane 6 rocket launches Sentinel-1D Earth-observation satellite to orbit (video)04.11.2025 - 4
Seven deaths possibly linked to malfunctioning glucose monitors02.12.2025 - 5
Indian rocket launches record-breaking BlueBird 6 smartphone satellite to orbit (video)23.12.2025 - 6
Sophie Kinsella, 'Confessions of a Shopaholic' author, dies at 55 after battle with cancer10.12.2025 - 7
The most effective method to Pick the Right Teeth Substitution Choice for You16.10.2023 - 8
U.S. to drop childhood vaccine recommendations as it looks to Denmark, Washington Post reports19.12.2025 - 9
Why Cannes Is the Ultimate New Year’s Eve Destination in the South of France’s Off-Season19.12.2025 - 10
Nigeria’s return to Windsor castle signals new era in UK economic partnership26.03.2026 - 11
US measles cases surpass 2,000, highest in 30 years: CDC30.12.2025 - 12
The Response to Independence from the rat race: Methodologies for Creating Financial momentum07.07.2023 - 13
Illegal entries into Germany halve over two years, border police say01.01.2026 - 14
James Webb Space Telescope finds strongest evidence yet for atmosphere around rocky exoplanet: 'It's really like a wet lava ball'11.12.2025 - 15
Holiday destinations for Creature Sweethearts01.01.1
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


































