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.
Recent announcements from three biotechnology and pharmaceutical companies reflect the increasing interest in and tractability of myotonic dystrophy for therapy development. Short summaries and links to the press releases are provided here.