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Making 2016 a Break-Through Year

MDF just kicked off the new year with our annual two-day strategic planning offsite. The planning team included DM researchers, board members and staff. Below are some highlights of our plans for 2016.

Advocacy: Raising the Community Voice 

The significant focus on Patient Focused Drug Development (PFDD) in Washington, DC has offered MDF the opportunity to bring the DM patient voice to drug development and review efforts, as well as initiatives to enhance patient and family care, via exciting new initiatives.

2016’s advocacy program includes the first-ever meeting of DM families and regulatory professionals from the Food and Drug Administration (FDA), European Medicines Agency (EMA) and other agencies. The meeting will take place during the 2016 MDF Annual Conference, September 15, 2016 and all interested registrants at this year’s conference will be invited to attend. Part of the preparatory work for this meeting will include surveys to both DM patients and caregivers in order to collect comprehensive information and community perspectives on treatment priorities and disease burden.

We will kick the 2016 advocacy effort off with a February webinar to share information on our congressional and grassroots strategies and directions for participating our Congressional Call-In for Rare Disease Day in February.

Improving Access and Standards of Care

 MDF is wrapping up an in-depth analysis of the state of DM care (from self-care to family and clinical care) in 2016 to identify barriers to accessing quality care, and identify care program priorities as we continue to build out our Care initiatives. MDF is also working with an international group of leading DM clinicians to develop the first-ever consensus-driven care recommendations. We are working in tandem with the American Academy of Neurology and its Evidence-Based Guideline project and expect to jointly release and disseminate both documents in late 2016. 

Building Community 

MDF will continue to build the DM community and create networks by sharing inspiring stories and DM science news, supporting grassroots events and fundraisers, expanding support groups, and hosting another MDF Annual Conference in fall 2016.

Driving Research: Every Body Counts Campaign

Participation in research by the DM community is essential if we are to achieve the breakthrough treatments we need. MDF will help researchers expand the number of community members engaged in DM studies and trials by sharing information throughout the year on how to participate in treatment trials, surveys and natural history data collection. We’ll also incentivize participation through an Every Body Counts campaign that will launch during Rare Disease Month. We need you. Every body counts when it comes to Care and a Cure.

Accelerating Drug Development

MDF will continue to carry out the objectives of MDF 3.0: Accelerating Drug Development, our 3-year plan to increase the number of DM therapy development efforts underway and enhance chances for drug development success. These include:

  • Strengthening capacity and infrastructure in DM clinical care to better understand the patient population and remove inconsistencies in care that can complicate potential therapy assessment
  • Deepening and strengthening the research and development bench with more investigators and more data
  • Expanding the drug development pipeline with additional industry and academic efforts
  • Providing incentives for investment in myotonic dystrophy therapy development

New Initiatives in 2016

Request for Applications to Develop DM Biomarkers 

MDF, in partnership with the Wyck Foundation, will issue a Request for Applications in Spring 2016 to develop or refine biomarkers needed to determine the efficacy of treatments in clinical trials.

Support the DM Clinical Research Network

There are currently six sites in the DM Clinical Research Network (DMCRN). The University of Rochester is the lead DMCRN site, with Drs. Richard Moxley and Charles Thornton as the DMCRN Lead PIs. MDF will continue to support the network as it conducts critical studies, works with the pharmaceutical companies on drug development, and brings new data and information to academic researchers and industry.

Expand Post-Doctoral Fellows Program

As part of our efforts to advance both Care and a Cure, MDF is expanding our post-doctoral Fellows program. To this end, MDF has entered into a strategic alliance with the Wyck Foundation, a UK-based non-profit that funds targeted DM-related projects. MDF is also investigating additional strategies to further expand the Fellows program internationally, provide additional training to Fellows and create formal networking opportunities to connect Fellows to each other, leading researchers and clinicians, and MDF community members.

Measure the Impact of DM 

MDF launched studies on DM burden of disease and population-based prevalence to help demonstrate the real scope and cost of this disease to individuals, families and society. This information will help build the case for industry investment in myotonic dystrophy therapies, and convince insurance companies and other payers to reimburse for eventual therapies. This information is also a key tool in our advocacy efforts with policymakers. 

Results of the first phase of the burden of disease study, which is being conducted in partnership with the Mayo Clinic and Optum Labs, should be available in mid-2016. The first phase of the prevalence study, which launched late last year via a grant to Dr. Nicholas Johnson at the University of Utah, should conclude in mid-2016 as well. More to come on those two projects.

Grow the Myotonic Dystrophy Family Registry

MDF continues to expand its DM registry, which is now one of the largest globally. In 2016, we will continue to explore how we can improve data collection and coordination across different DM data sets, and how a potential partnership with the University of Rochester registry might work. 

2016 is going to be another banner year. We look forward to working with you to make a major impact on Care and a Cure.





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