Posted by Michael Wonder on 08 May 2024
Avidity Biosciences receives FDA breakthrough therapy designation for delpacibart etedesiran (AOC 1001) for treatment of myotonic dystrophy type 1
8 May 2024 - Delpacibart etedesiran data from MARINA-OLE showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data.
Avidity Biosciences today announced that the US FDA has granted breakthrough therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1.
