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Care and a Cure in 2015: How You Can Help








November 2014

Dear MDF Community Member:

We write to you this year with an unprecedented sense of joy and urgency. The Myotonic Dystrophy Foundation (MDF) remains wholeheartedly focused on supporting and driving the research, resources and community capacity needed to achieve the first clinical trials for myotonic dystrophy treatments, while providing comprehensive support and education to families living with this disease.

We are delighted that the first clinical trials are underway. MDF has played an important role to date in supporting academic and industry partners involved in treatment development. Going forward, we will concentrate our strategic efforts on developing multi-year advocacy initatives to:

  • Sustain and grow the drug development pipeline;
  • Educate regulatory, research and other federal agencies that make decisions about drug approvals and research funding for myotonic dystrophy;
  • Represent the myotonic dystrophy community to these key stakeholders who help determine the future course of this disease; and
  • Raise awareness about myotonic dystrophy to shorten the diagnostic odyssey and improve patient care.

Our objectives for these initiatives are aggressive and ambitious:

  • Expand annual federal research funding for myotonic dystrophy from $11 million to over $30 million;
  • Increase industry commitment to treatment development for myotonic dystrophy from $0.5 billion to over $2 billion;
  • Promote best possible drug review and approval environment; and
  • Ensure access to treatments once they enter the market.

We will undertake this work over the next three years while maintaining and growing the support groups, webinars, events and other community and research programs that help improve the quality of life of people living with this disease.

We have suffered the devastating loss of too many family members, and we must redouble our efforts to change the prospects and future for people living with myotonic dystrophy now.

This moment is critical, and we need your help. Please consider making a gift to MDF this year to help us achieve our urgent objectives.

Your ongoing support of the Myotonic Dystrophy Foundation has helped bring us to the truly hopeful moment we celebrate today. Thank you, on behalf of affected families around the world, for joining us in this important work. We could not do all that we do without you, and we continue to be deeply grateful for your commitment.



Jeremy Kelly, Chairman

Molly White, Executive Director






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