
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
Wolf Bites Woman in Shocking Attack at Busy Shopping Center31.03.2026 - 2
Mexican Woman Accused of Assaulting Partner With Belt After He Refused Sex, Police Say28.03.2026 - 3
Strength training is crucial after menopause. How to make the most of your workouts10.01.2026 - 4
One month of war on Iran cost Arab countries up to $194bn: UNDP31.03.2026 - 5
Gen Z workplace stereotypes were TV’s favorite punchline in 202530.12.2025
Ähnliche Artikel
Syrian army says recent drone attacks targeted its bases near Iraq, most shot down30.03.2026
Tributes pour in for James Ransone, 'The Wire' actor who died at 4622.12.2025
Artemis 2 astronauts arrive at Kennedy Space Center ahead of NASA's historic launch around the moon27.03.2026
‘Everybody Loves Raymond: 30th Anniversary Reunion’ premiere date: How to watch, channel, time, cast and more22.11.2025
As cases of a rare, deadly infection rise, doctors worry fewer teens will get vaccinated11.01.2026
Did we start the fire? A 400,000-year-old hearth sparks new questions about human evolution10.12.2025
Health officials report 14 Legionnaires' disease cases in Florida, gym connection suspected05.12.2025
Hamas hands over another body in the Gaza Strip13.11.2025
The Most Compelling Books of the 10 years05.07.2023
Ford Is Using a Chinese-Built Van to Fight Europe’s EV Price War28.03.2026














