In this study, researchers at Baylor College of Medicine and their collaborators focused on the DM1 heart problems testing a novel approach to restore normal function. The study appeared in The ...

RBC noted that while Avidity (RNA) is ahead on myotonic dystrophy type 1, or DM1, Dyne (DYN) may catch up with its Fab-ASO ...

Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations ...

This marks the first such designation for a DM1 treatment in Japan and follows similar recognitions in the U.S. and Europe. According to InvestingPro data, the company maintains a strong financial ...