New hope for duchenne: targeted drug trial aims to slow muscle decline
Disease control
Not yet recruiting
This study tests an experimental drug called AOC 1044 (delpacibart zotadirsen) in 70 boys aged 7 to 16 with Duchenne muscular dystrophy who have a specific genetic change (exon 44 skipping). The drug is given through a vein and aims to improve muscle function, such as how fast th…
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 15, 2026 11:57 UTC