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.
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.
Dr. Vincent Dion awarded $250,000 grant to pursue gene editing technology development to find a cure for DM.
The Myotonic Dystrophy Clinical Research Network (DMCRN) sites are currently conducting a critically-important research study designed to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies.
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