Safety, tolerability, and pharmacokinetics of casimersen in patients with <scp>D</scp>uchenne muscular dystrophy amenable to exon <scp>45</scp> skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial

Abstract

Casimersen was well tolerated in participants with DMD amenable to exon 45 skipping. Most TEAEs were mild, nonserious, and unrelated to casimersen. Plasma exposure was dose proportional with no suggestion of plasma accumulation. These results support further studies of casimersen in this population.