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04/28/2016 - 4:29am

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. 

04/19/2016 - 1:06pm

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.

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.

05/18/2016 - 8:55am

MDF's Interim Chief Science Officer, Dr. John Porter, brings a wealth of experience to MDF as a former academic researcher, program director at the National Institutes of health (NIH) and Chief Executive Officer of a patient advocacy organization. John offered his thoughts recently on the importance of patient advocacy in drug development.

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.


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