P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study

Autor: Erin O’Rourke, Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Natalie F. Reash, Megan Iammarino, Lindsay Alfano, Sarah Lewis, Kathleen Church, Richard Shell, Rachael A. Potter, Danielle Griffin, Mark J. Hogan, Shufang Wang, Stefanie Mason, Eddie Darton, Louise Rodino-Klapac
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Genetics in Medicine Open, Vol 1, Iss 1, Pp 100342- (2023)
Druh dokumentu: article
ISSN: 2949-7744
DOI: 10.1016/j.gimo.2023.100342
Databáze: Directory of Open Access Journals