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April 19, 2016

The FDA will have the final say on whether therapies developed to treat myotonic dystrophy (DM) are approved for US patients, and they want to hear from DM patients to help with their decision-making. Join the DM Patient-Focused Drug Development Meeting and let the FDA know what it is like to live with DM, and what you want from upcoming therapies.

April 19, 2016

Dr. Laurence Mignon, Director of Clinical Development at Ionis Pharmaceuticals in Carlsbad, CA, and Dr. Charles Thornton, neurologist and a principal investigator at the University of Rochester, reported on the status of the Phase I and Phase I/II clinical trials for IONIS -DMPKrx, a potential DM therapy, at the annual meeting of the American Academy of Neurology in Vancouver, BC this week. Dr. Mignon shared the results of the presentations with MDF so we could report out to the MDF community via the Dispatch

April 19, 2016

A new study by a multi-disciplinary, international team has shed light on the molecular changes at the root of DM-related heart dysfunction, the second leading cause of death for DM patients.

April 2, 2016

Researchers from the University of Costa Rica and the University of Glasgow teamed up to investigate the DNA mutation causing myotonic dystrophy type 1 (DM1). They found that polymorphism in the MSH3 mismatch repair gene is associated with the levels of somatic instability of the expanded CTG repeat in the blood DNA of myotonic dystrophy type 1 patients. 

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