Overview of eteplirsen (AVI-4658/Exondys51) clinical trials

Trial publications Phase I local treatment (NCT00159250) Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy • Kinali M, Arechavala-Gomeza V, Feng L, et al. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. The Lancet. Neurology 2009;8:918-28 • Cirak S, Feng L, […]