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AOC 1044 demonstrated favorable safety and tolerability. Avidity also announced delpacibart zotadirsen as the approved international nonproprietary name of AOC 1044, abbreviated as del-zota.
Statistically significant increase of 25% of normal in dystrophin production and restored total dystrophin up to 54% of normal with delpacibart zotadirsen 5 mg/kg at four months
Diana Castro, M.D., Board Certified Neurologist and Neuromuscular Physician, Founder and Director Neurology and Neuromuscular Care Center, Founder and Director Neurology Rare Disease Center and EXPLORE44 trial program investigator, said, "These del-zota data are very encouraging as children and adults living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping are still in need of targeted treatment options to address this debilitating disease."
Posted In: RNA