- A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping — Recruiting • Phase III • NCT07587242.
- New drug tested for Duchenne muscular dystrophy in patients whose genetic mutations can be treated by skipping exon 44.
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A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping Conditions: Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease, Musculoskeletal Diseases, Neuromuscular Diseases (NMD), Nervous System Diseases, Genetic Diseases, X-Linked, Hereditary, Neonatal Disease, Duchene Muscular Dystrophy, Congenital, DMD Interventions: AOC 1044, Placebo Lead Sponsor: Avidity Biosciences, Inc. Planned Enrollment: 70 participants