DM1

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 Myotonic 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.

Myotonic Medical School Roadshow

Myotonic 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.

Decline in Daily and Social Activities in DM1

Daily activities and social participation are assessed in a longitudinal study of DM1.

New Research Study on Cognitive Function and Neuroimaging CDM

Dr. Melissa Dixon at the University of Utah Department of Pediatrics is conducting a study to learn more about how congenital myotonic dystrophy (CDM) and childhood-onset myotonic dystrophy affect thinking, memory, attention, brain function, and how these processes change over time.

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