Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

Sarepta Reports Second Death of Patient Using Its Gene Therapy

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for a rare muscle disorder.

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun 15, 2025

Sarepta reports second patient death after treatment with Duchenne gene therapy

The company said it had paused shipments of the therapy, called Elevidys, for patients who can no longer walk.

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun 15, 2025-

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne | Sarepta Therapeutics, Inc.

The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators - Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved

Data on Duchenne Muscular Dystrophy Treatment From MDA 2025

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North Star Ambulatory Assessment, time to rise from floor, and 10-m walk/run compared with external control groups over 2 years of treatment.