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Help the FDA Review Potential DM Therapies


Don’t miss this once-in-a-lifetime opportunity to tell the FDA what it is like to live with myotonic dystrophy, and what your priorities are for DM treatments.

MDF is bringing all interested MDF conference attendees to the first-ever patient-focused drug development meeting with the FDA during this year’s conference. Learn more about the DM Patient-Focused Drug Development Meeting here

Need information on the 2016 MDF Annual Conference? Click here.
 

Molecular Changes Lead to Cardiac Dysfunction in DM

 
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. Read more here.
 

Ionis Pharmaceuticals Reports on Studies

 
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. Read the rest here.
 

New Findings on the DM1 DNA Mutation 


Researchers from the University of Costa Rica and the University of Glasgow found that a 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. Read more here.
 

MDF Welcomes Dr. John Porter as Interim Chief Science Officer

Dr. John Porter, former Program Director of Neuromuscular Disease at the National Institute of Neurological Disorders and Stroke (NINDS), a component of the National Institutes of Health (NIH), has joined MDF as Interim Chief Science Officer. Dr. Sharon Hesterlee will remain active on our Scientific Advisory Committee while taking on a new role as EVP of Patient Advocacy and Public Affairs at Bamboo Therapeutics. Read more here.

Upcoming Support Group Meetings:

4/30 - Seattle, WA 
5/3 - Ottawa, Canada
5/18 - Portland, OR
5/21 - New Orleans, LA
5/21 - Denver, CO
5/21 - Indianapolis, IN
5/21 - Atlanta, GA
5/21 - Northern CA
5/22 - Orange County, CA
5/22 - Dallas, TX
Have you visited the Digital Academy lately? We've got over a hundred videos of past conferences, family stories, science talks, and more! Watch our Facebook page, too, to see videos we are featuring!
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Phone: (415) 800-7777
Email: info@myotonic.org
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