| Title: |
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management |
| Authors: |
Shieh, Perry B.; Proud, Crystal; Diamond, Tamir; Chapin, Catherine A.; Ahmad, Jawad; Salama, Alan D.; Bönnemann, Carsten G.; Soslow, Jonathan; Byrne, Barry J.; Veerapandiyan, Aravindhan; Brandsema, John F.; Matesanz, Susan; Samelson-Jones, Benjamin J.; Wilkins, Benjamin J.; Gerber, Marianne; Godwin Wild, Kara E.; Mason, Stefanie; Asher, Damon; McDonald, Craig M.; Mendell, Jerry R. |
| Contributors: |
Sarepta Therapeutics |
| Source: |
Human Gene Therapy ; ISSN 1043-0342 1557-7422 |
| Publisher Information: |
SAGE Publications |
| Publication Year: |
2026 |
| Description: |
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 |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1177/10430342261423168 |
| Availability: |
https://doi.org/10.1177/10430342261423168; https://journals.sagepub.com/doi/pdf/10.1177/10430342261423168; https://journals.sagepub.com/doi/full-xml/10.1177/10430342261423168 |
| Rights: |
https://creativecommons.org/licenses/by/4.0/ ; https://journals.sagepub.com/page/policies/text-and-data-mining-license |
| Accession Number: |
edsbas.27325463 |
| Database: |
BASE |