Published on 4 Mar 2024 on Benzinga
Monday, Avidity Biosciences Inc RNA revealed new long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE) trial showing reversal of disease progression in myotonic dystrophy type 1 (DM1) patients across multiple endpoints compared to END-DM1 natural history data.
These are the same key endpoints used in the global Phase 3 HARBOR trial for DM1.