FDA Grants Rare Pediatric Disease Designation to Duchenne Agent AOC 1044 for Mutations Amenable to Exon 44 Skipping
According to a recent announcement, the FDA has granted rare pediatric disease designation to AOC 1044, Avidity Biosciences’ investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients living with mutations amenable to exon …