Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.

Jerry R MendellZarife SahenkKelly J LehmanLinda P LowesNatalie F ReashMegan A IammarinoLindsay N AlfanoSarah LewisKathleen ChurchRichard ShellRachael A PotterDanielle A GriffinMark HoganShufang WangStefanie MasonEddie DartonLouise R Rodino-Klapac

Published in: Muscle & nerve (2023)

Gene transfer therapy with delandistrogene moxeparvovec treatment is well tolerated, with a favorable safety profile. Functional improvements are sustained through 4 years, suggesting delandistrogene moxeparvovec may positively alter disease progression.

Keyphrases

duchenne muscular dystrophy
gene therapy
clinical trial
study protocol
genome wide
copy number
phase iii
randomized controlled trial
gene expression
mesenchymal stem cells
double blind