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April 28, 2016

This Mother’s Day and Father’s Day, MDF reflects on what it means to be a family. Myotonic dystrophy is a family disease, in every sense of the word. Not only does it run in families, it takes a family to meet the challenges DM poses every day. We will be sharing stories of courage and hope from families like yours from now until Father’s Day to inspire giving. 

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 last month.

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.


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