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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.

June 7, 2016

A former MDF Fellow, Dr. Yao Yao, has made a breakthrough in understanding how muscle stem cells are directed to aid muscle regeneration. Read about his important research here.

May 18, 2016

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.

May 18, 2016

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.

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