MDF has had a busy few months of activity in Washington, DC, continuing our efforts to secure research funds, engage lawmakers and make our presence known on Capitol Hill in 2019.
SAN FRANCISCO, CA (May 2, 2019): The Myotonic Dystrophy Foundation (MDF) is pleased to announce the publication of the first-ever Consensus-based Care Recommendations for Congenital and Childhood-onset Myotonic Dystrophy Type 1 and Myotonic Dystrophy Type 2.
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.