FDA Limits Duchenne Gene Therapy After Two Teens Die of Liver Failure

Op-Ed: New York puts a price on children’s lives (and refuses to pay it)

New York is known to be a high-tax and higher spending state. So, it’s curious that it is attempting to violate federal law and deny dying children the gene therapy

FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk

Risk of “acute, serious, and life-threatening” heart inflammation is now on the label of Sarepta's gene therapy for Duchenne muscular dystrophy.

FDA Limits Duchenne Gene Therapy After Two Teens Die of Liver Failure

Key Takeaways

FDA limits Elevidys use; Nxera to lay off staff

Elevidys now has a “boxed” warning and is no longer available for Duchenne patients who can’t walk. Elsewhere, Nuvalent and Zymeworks unveiled data for targeted cancer drugs.

Liver Deaths Prompt FDA Boxed Warning for Elevidys for DMD

The FDA is adding a Boxed Warning label to delandistrogene moxeparvovec-rokl, a gene therapy for Duchenne muscular dystrophy, after reports of fatal liver injuries. Medscape Medical News Source link : https://www.medscape.com/viewarticle/liver-deaths-prompt-fda-boxed-warning-elevidys-dmd-2025a1000w5l?src=rss Author : Publish date : 2025-11-18 21:11:00 Copyright for syndicated content belongs to the linked Source. The post Liver Deaths Prompt FDA…

Sarepta adds boxed warning to Elevidys about risk of liver disease

In addition, Elevidys’s indication for non-patients has been removed, and the gene is now indicated only to treat ambulatory patients four years of age and older with Duchenne muscular dystrophy.