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MDF Publishes First-ever CDM and DM2 Clinical Care Recommendations

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.

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.

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.

MDF Medical School Roadshow

MDF designed this volunteer initiative to educate the next generation of medical professionals about myotonic dystrophy in order to improve clinical care and shorten the diagnostic odyssey. We need you!

Potential for Genome Editing in Neuromuscular Disease

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

New Perspectives on Respiratory Involvement in DM1

Respiratory dysfunction in DM1 draws new attention.


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