| Title: |
Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial |
| Authors: |
Mendell, Jerry R.; Sahenk, Zarife; Lehman, Kelly J.; Lowes, Linda P.; Reash, Natalie F.; Iammarino, Megan A.; Alfano, Lindsay N.; Lewis, Sarah; Church, Kathleen; Shell, Richard; Potter, Rachael A.; Griffin, Danielle A.; Hogan, Mark; Wang, Shufang; Mason, Stefanie; Darton, Eddie; Rodino‐Klapac, Louise R. |
| Contributors: |
Nationwide Children's Hospital; Parent Project Muscular Dystrophy; Sarepta Therapeutics |
| Source: |
Muscle & Nerve ; volume 69, issue 1, page 93-98 ; ISSN 0148-639X 1097-4598 |
| Publisher Information: |
Wiley |
| Publication Year: |
2023 |
| Collection: |
Wiley Online Library (Open Access Articles via Crossref) |
| Description: |
Introduction/Aims Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Long‐term delandistrogene moxeparvovec microdystrophin protein (a shortened dystrophin that retains key functional domains of the wild‐type protein) expression may positively alter disease progression in patients with DMD. We evaluated long‐term safety and functional outcomes of delandistrogene moxeparvovec in patients with DMD. Methods An open‐label, phase 1/2a, nonrandomized controlled trial (Study 101; NCT03375164) enrolled ambulatory males, ≥4 to |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1002/mus.27955 |
| Availability: |
https://doi.org/10.1002/mus.27955; https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27955 |
| Rights: |
http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| Accession Number: |
edsbas.A3D80906 |
| Database: |
BASE |