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Care and a Cure -- We Need Your Help

It’s been a very impactful year for myotonic dystrophy and MDF, and we’re excited about what’s ahead in the search for treatments and a cure for DM.

In 2013, MDF—the world’s largest myotonic dystrophy patient organization—sponsored a range of initiatives to accelerate research into DM treatments:

  • Launched the Myotonic Dystrophy Family Registry at the request of the research community, now the largest DM patient registry in the US
  • Supported the DM Clinical Research Network, the first clinical research network for myotonic dystrophy, led by the University of Rochester in partnership with Stanford University, Ohio State University, the University of Kansas and the University of Florida
  • Initiated the DM & Pregnancy Study to study effects of DM in pregnant women
  • Provided recruitment and communications support for academic research studies
  • Networked and created critical partnerships with key DM stakeholdersincluding relationships with pharmaceutical and biotech companies, FDA representatives and other corporate and government interests who are part of the U.S. drug approval process

MDF has a critical and unique role to play in the drug development and approval process.  In the words of Dr. John Day, Director of the Stanford Neuromuscular Disorders Program,

 “MDF is critical [in the drug research and development process].  We need MDF to both educate patients so they can understand what’s going on, and to help patients and institutions communicate [and interact].  It’s got to be a partnership and it can’t be done by the institutions themselves.”

MDF has also expanded its support and resources to people living with DM. This year we launched several new support programs, including the Living with DM monthly webinar series. We also initiated a Spanish language translation of the MDF Toolkit and introduced a robust grassroots campaign to bolster fundraising and support programs across the country. 

With the first clinical trials for myotonic dystrophy treatments scheduled for 2014, next year will be critical for DM research. We need your continued partnership as never before to ensure that the unprecedented progress toward treatment identification and approval continues. 

Your gift will benefit research efforts far beyond DM. Because research to develop treatments targeting the DM mutation is leading to significant breakthroughs in other neuromuscular and genetic diseases, the opportunity for your gift to have an impact has never been greater.

Please consider making a new or renewed investment in MDF. You’ll be supporting our work to advance research, and significantly leveraging your investment beyond DM to genetic and neuromuscular research overall. In the meantime, you’ll be ensuring that critical support and education services continue to be available to people living with DM, those newly-diagnosed and those confronting new stages in disease progression. 

Our donors are essential to the success of our work, and we are deeply grateful for your support. Thank you.






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