
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
Internet goes (cocoa) nuts: The funniest reactions to 12 tonne theft of KitKat bars30.03.2026 - 2
'No middle ground' for tackling antisemitism after Bondi Beach mass shooting, deputy FM Haskel says14.12.2025 - 3
'Set up an Army Radio station at President’s Residence': Source close to Katz slams Herzog13.11.2025 - 4
RSF attack on Sudan’s South Kordofan kills at least 14, including children29.03.2026 - 5
Pocket-Accommodating Jeep Wrangler Buying Guide for Seniors06.11.2023
Ähnliche Artikel
How to get tickets to Jay-Z’s sold out Yankee Stadium shows25.03.2026
Manual for Conservative SUVs For Seniors05.06.2024
6 Popular Ladies' Aromas On the planet22.09.2023
Nikki Glaser has been testing out Golden Globes jokes. There's one nobody wants to hear06.01.2026
More loons are filling Maine's lakes with their ghostlike calls13.12.2025
Muslim Brotherhood stole half a billion dollars in Gaza donations, Arab sources reveal17.11.2025
Famous Restroom Beautifying Styles For 202405.06.2024
IDF continues counterterrorism operations in Gaza Strip, including destroying terror tunnels27.03.2026
When preventable infections turn deadly behind bars | The Excerpt20.11.2025
A top Marine shares his secrets to keeping fit at 5029.11.2025














