Work

P47 ENVISION, a phase 3, randomized trial evaluating the safety and efficacy of delandistrogene moxeparvovec in Duchenne muscular dystrophy: study design

Year: 2023

Type: conference-abstract

Source: Neuromuscular Disorders

DOI: 10.1016/j.nmd.2023.07.028

Authors: F. Muntoni, E. Mercuri, C. McDonald, I. Desguerre, M. Tulinius, C. Proud, M. Furgerson, A. Murphy, C. De Ford, T. Feng, C. Reid, C. Wandel, N. Shelton

Institutions: Great Ormond Street Hospital, University College London, Agostino Gemelli University Polyclinic, Istituti di Ricovero e Cura a Carattere Scientifico, UC Davis Health, Hôpital Necker-Enfants Malades, Université Paris Cité, University of Gothenburg, Children's Hospital of The King's Daughters, Sarepta Therapeutics (United States), F. Hoffmann-La Roche AG, Roche (United Kingdom)

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4387309671

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