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05/18/2016 - 9:14am

For the first time, researchers have a means of targeting RNA in living cells. There is potential for this approach to edit the CTG and CTTG expansions in DM1 and DM2. Read the article from MDF to learn more.

05/11/2016 - 6:26am

Dr. Matt Disney brings an unusual and increasingly valuable skill to therapy development for DM—he’s a chemist. The NIH has recently awarded two research grants in support of Dr. Disney’s research. Read this article to find out how his work leverages the latest advances in RNA biology to target the unique disease mechanisms of DM.

05/11/2016 - 5:55am

Dr. Guillaume Bassez and a large team in France and Canada recently published an analysis of gender and its impact on the DM1 phenotype. Read about their findings in this article.

04/19/2016 - 7:53pm

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.

04/19/2016 - 7:23pm

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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