Defective insulin signaling activation may underlie skeletal muscle wasting in DM1 and DM2.
Preclinical proof of concept is published for a small molecule strategy targeting toxic DM1 repeat-expanded RNA.
A recent study implicates RNAi driven by CUGexp as an independent mechanism in altered gene expression and pathogenesis of DM1.
The Myotonic Dystrophy Foundation is currently planning a workshop to address CNS involvement in DM1, DM2 and CDM, to be held on September 12, 2019, in conjunction with the 2019 MDF Annual Conference, September 13-14, in Philadelphia, PA.
Nationwide Children’s Hospital and Ohio State University have operated a 5-day myology training course for the last seven years. The course includes common lectures in the mornings and separate clinical and laboratory tracks in the afternoons.