Efficacy and safety of delandistrogene moxeparvovec for children with Duchenne muscular dystrophy: A Systematic Review

OSF Preprints (OSF Preprints) 2026 Preprint

Authors: Carolina de Oliveira Cruz Latorraca, Patrícia do Carmo Silva Parreira, Verônica Colpani, Rachel Riera, Roberta Borges Silva, Cinara Stein, Isabela Porto de Toledo, Camila Monteiro Cruz, Rafael Leite Pacheco
Cited by: 0

Abstract

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular degenerative disease with onset in early childhood. Delandistrogene moxeparvovec (Elevidys®) is a single-dose gene therapy, aiming to boost microdystrophin expression in skeletal muscles. Thus, the systematic review aimed to assess the efficacy and safety of Elevidys® for children with DMD.

Fields of Study & Subjects

Muscle Physiology and Disorders Neurogenetic and Muscular Disorders Research Virus-based gene therapy research

Publication details

Journal / source
OSF Preprints (OSF Preprints)
Publication date
28 January 2026
Language
EN
Document type
Preprint