424PDelandistrogene moxeparvovec micro-dystrophin expression and safety in 3–4-year-olds with Duchenne muscular dystrophy in ENDEAVOR and ENVOL studies
Year: 2025
Type: article
Source: Neuromuscular Disorders
Authors: C. McDonald, J. Mendell, E. Mercuri, A. Gangfuss, A. Nascimento, I. Desguerre, C. Zaidman, L. Servais, P. Thrasher, M. Vivien, K. Ding, A. Murphy, B. Zhang, K. Wonde, P. Pordeli, L. Rodino-Klapac
Institutions: UC Davis Health, Nationwide Children's Hospital, Agostino Gemelli University Polyclinic, Istituti di Ricovero e Cura a Carattere Scientifico, Essen University Hospital, University of Duisburg-Essen, Hospital Sant Joan de Déu Barcelona, Hôpital Necker-Enfants Malades, Université Paris Cité, Washington University in St. Louis, University of Liège, University of Oxford, Centre Hospitalier Universitaire de Liège, Sarepta Therapeutics (United States), Roche (United Kingdom), Roche (Canada), F. Hoffmann-La Roche AG
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: closed
OpenAlex ID: W4414824201