DMD – THERAPY: P.283 Long-term safety and efficacy of golodirsen in male patients with Duchenne muscular dystrophy amenable to Exon 53 skipping

Abstract

Golodirsen is a synthetic phosphorodiamidate morpholino oligomer designed to restore the mRNA reading frame in patients with DMD gene mutations amenable to exon 53 skipping. We report the safety and efficacy of long-term golodirsen treatment in this first-in-human, phase 1/2, two-part, multicenter trial. Twelve patients from part 1 (randomized, 12-week dose-titration phase; golodirsen [n = 8], placebo [n = 4]) plus 13 additional patients with genotypically confirmed DMD amenable to exon 53 skipping therapy were included in part 2 (open label).