Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-<i>κ</i>B Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

This first report of edasalonexent oral administration for one week in male pediatric patients with Duchenne muscular dystrophy showed that treatment was well tolerated and inhibited NF-kB pathways.