Research

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 CUG_exp as an independent mechanism in altered gene expression and pathogenesis of DM1.

MDF will host a Workshop on CNS involvement in DM and the development of outcome measures for CNS-targeting therapies on September 12, 2019, in conjunction with the MDF Annual Con

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.

Disorders of sleep and breathing are well characterized for DM1, but what about DM2?

A new review looks at the opportunities and hurdles for genome editing in neuromuscular disease.