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Research

Linking DM Molecular Events to Insulin Resistance and Muscle Atrophy

Defective insulin signaling activation may underlie skeletal muscle wasting in DM1 and DM2.

Targeting Toxic RNA in DM1 with a Small Molecule Drug

Preclinical proof of concept is published for a small molecule strategy targeting toxic DM1 repeat-expanded RNA.

Potential Involvement of RNAi Mechanisms in DM1

A recent study implicates RNAi driven by CUGexp as an independent mechanism in altered gene expression and pathogenesis of DM1.

CNS and Myotonic Dystrophy: Scientific Premise and Potential Targets Workshop

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.

Annual Nationwide Children’s Hospital/OSU Myology Course

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.

Pharma/Biotech Updates

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 a $250,000 MDF Grant to Pursue Cure for DM

Dr. Vincent Dion awarded $250,000 grant to pursue gene editing technology development to find a cure for DM.

Participants Needed for Important DM Research Study

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.

Sleep and Disordered Breathing in DM2

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

Potential for Genome Editing in Neuromuscular Disease

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

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