DMD and Duchenne muscular dystrophy: Currently undergoing a Phase 1b/2a open-label study named FORWARD-53, this drug aims to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in DMD patients (NCT04906460).Phase 1b/2a Part A proof-of-concept trial in boys with DMD amenable to exon 53 skipping showed promising results, with all treatment-related adverse events being mild and the treatment appearing safe and well-tolerated (GlobeNewswire, 2022).