Programs and Events

In 2016, MDF’s Warmline received many phone calls from myotonic dystrophy community members who struggled to complete the application process for federal and state disability benefits, and who needed guidance on how to navigate the complex application process.

The myotonic dystrophy (DM) community has a strong champion in singer-songwriter Eric Hutchinson. As part of his long-time efforts to support Care and a Cure for myotonic dystrophy, Eric is offering one-of-a-kind fan activities and memorabilia in a new pledge campaign, and a portion of the proceeds will be donated to MDF.

Congratulations to our Community Leadership Award winners: Loraine Dressler, Carolyn Valek and Eric & Taylor Jensen! Click here to learn more about the amazing work they do for Care and a Cure.

MDF held the first myotonic dystrophy Patient-Focused Drug Development meeting with key senior leadership from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference. The meeting, the first Externally-Led PFDD meeting and the largest PFDD meeting held to date with over 200 attendees in the room and online, is helping change the way the FDA understands DM, the DM community and the approach to regulatory review for DM therapies. 

The Jensen family hosts an incredible crawfish boil each summer to support Care and a Cure. This year’s boil was bigger than ever with San Diego Harley-Davidson as a partner. To date, this event has raised nearly $100,000! Read more here.

Our Staff recently attended the annual BIO convention, the meeting and partnering place for the biotechnology and pharmaceutical industry. BIO is about opportunities—opportunities for learning and working together to foster new drug development. MDF engaged key industry leaders in discussions of therapy development in DM, exchanging information on existing DM drug discovery and development, as well as opportunities for launching new DM programs.

MDF and its international team of clinical care partners just met in Miami to draft consensus-based Care Considerations for doctors, pharmaceutical companies and federal regulators reviewing potential therapies. Read more in this article.

Researchers from the University of Costa Rica and the University of Glasgow teamed up to investigate the DNA mutation causing myotonic dystrophy type 1 (DM1). They found that 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.