1). Like many other muscular dystrophies ... ARTHEx also has ATX-01 in preclinical testing for myotonic dystrophy type 2 (DM2). “Although DM2 is caused by a different mutation, it similarly ...

The analysis indicates that in 2023, the US had approximately 43,255 diagnosed cases of Myotonic Dystrophy Type 1 and 10,815 cases of Myotonic Dystrophy Type 2. This data highlights a significant ...

EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Myotonic Dystrophy Type 1 (DM1) arises from an expanded microsatellite CTG repeat ... We have generated constructs composed of APEX2 fused to MBNL1/2, which localizes the APEX2 protein to the RNA foci ...