Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management

Human Gene Therapy 2026 Open access

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Authors: Perry Shieh, Crystal Proud, Tamir Diamond, Catherine A. Chapin, Jawad Ahmad, Alan D. Salama, Carsten G. Bönnemann, Jonathan H. Soslow, Barry J. Byrne, Aravindhan Veerapandiyan, John F. Brandsema, Susan Matesanz
+8 more B. J. Samelson-Jones, Benjamin J. Wilkins, Marianne Gerber, Kara E. Godwin Wild, Stefanie Mason, Damon R. Asher, Craig M. McDonald, Jerry R. Mendell
DOI: 10.1177/10430342261423168
Cited by: 3
References: 34

Abstract

Acute liver injury (ALI) is a recognized adverse event with adeno-associated virus (AAV)-based gene therapies, including delandistrogene moxeparvovec, an AAVrh74-based gene therapy for Duchenne muscular dystrophy. Progression of ALI to acute liver failure (ALF) is rare. In clinical trials, ALF was defined as an international normalized ratio ≥1.5 with encephalopathy and/or ascites, occurring

Fields of Study & Subjects

Muscle Physiology and Disorders Virus-based gene therapy research Neurogenetic and Muscular Disorders Research

Publication details

Journal / source
Human Gene Therapy
Publisher
SAGE Publications
Volume
37
Issue
5-6
Pages
262–279
Publication date
12 February 2026
Language
EN