Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kappaB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

Finanger E, Vandenborne K, Finkel RS, et al. Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kappaB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy. Journal of neuromuscular diseases 2019;6:43-54   Abstract BACKGROUND: Edasalonexent is an orally administered small molecule designed to inhibit NF-kappaB, which is activated from infancy in Duchenne muscular dystrophy and is […]