The 2014 MDF Annual Conference, which occurred Friday, September 12 and Saturday, September 13, was a singular success, featuring oversold registration, an unparalleled number of researchers, pharma and biotech partners and federal agency representatives, and a number of impressive programmatic ‘firsts.’

If you couldn't join us this year, you can view recordings of the sessions by topic area in the Digital Academy or via links in the 2014 MDF Annual Conference Agenda.

Conference Announcement: $3-5mm Research and Advocacy Investment

Advocacy, and specifically driving DM drug development and access, will continue to be a key and growing focus of MDF's work over the next three to five years. As MDF Chairman Jeremy Kelly explained during this year's conference, MDF will be making a $3-5 million investment.

It's aggressive and urgent objectives include:

• Deepening the academic bench focused on myotonic dystrophy to drive more DM discovery;
• Expanding the drug development pipeline with additional pharmaceutical partners and translational research;
• Expediting the drug approval process via a targeted advocacy effort with federal agencies and legislators;
• Influencing patient access to eventual treatments through outreach and education with the payer community.

Look for updates and more information on this advocacy initiative in the coming months.

Friends and Heroes Reception

MDF kicked off the conference with our first annual Friends and Heroes Awards Reception, hosting over 150 US Senators and Representatives, researchers, industry partners and philanthropists from the MDF community. The event, which honored MDF community leaders who have made significant contributions in support of Care and a Cure for myotonic dystrophy, was held in the beautiful Kennedy Caucus Room in the Russell Senate Office Building on Capitol Hill.

The Foundation was deeply honored to present the following awards:

• Community Leadership Award to MDF Board member and Senator Tim Kaine
• Humanitarian Award to MDF philanthropist Addy Loudiadis
• Institutional Partner Award to the National Institutes of Health, Dr. Alan Guttmacher accepting
• Outstanding Achievement in DM Research to Drs. Richard Moxley III and Charles Thornton, University of Rochester
• Outstanding Contribution to DM Clinical Care to Dr. John Day, Stanford University
• Outstanding Contribution to Rare Disease Health Policy to:
  • Senator Sherrod Brown, D-OH
  • Senator Amy Klobuchar, D-MN
  • Representative Fred Upton, R-MI
  • Representative Henry Waxman, D-CA
  • Senator Roger Wicker, R-MS

Lesli Rotenberg , General Manager of Children and Family Programming and Senior Vice President of Marketing and Communications at PBS, was a particularly gracious emcee, and community members Sarah Berman, Elizabeth Conte, Jeremy and Erica Kelly, Dr. Woodie Kessel, Larry and Hunter Lord, Dr. Richard Lymn, Teresa and Doug Schmertman, Todd Stone, Saurabh Rai and Marlo and Dr. Glenn Wiggans presented the awards.

For photos of the event, visit the MDF Facebook Page to see the conference photo album.

Grassroots Advocacy Training and Capitol Hill Visit

Advocacy was the theme of the conference and is some of the most critical work the MDF community is undertaking right now. The nation's capital provided the perfect backgrop for conference attendees to gain first-hand experience in advocating for Care and a Cure.

75 MDF community advocates attended a Friday morning briefing and then boarded buses to Capitol Hill to meet with Senators, Representatives and their staffs from more than 25 states. They educated congressional leadership and staffers about myotonic dystrophy and the importance of the MD-CARE Act, which was reauthorized just six days later !

MDF Science Workshop

MDF hosted its first science workshop, including more than 50 senior representatives from industry, the National Institutes of Health, the U.S. Food and Drug Administration, and the academic research community in a discussion entitled Measuring Drug Effects in Clinical Trials: Endpoints and Biomarkers. The workshop, the first of its kind in the US, featured opening remarks from Dr. Alan Guttmacher, Director of the National Institute for Child Health and Human Development at the NIH, and formerly of the Human Genome Project, followed by presentations from Drs. Chad Heatwole, Andy Berglund, Eric Wang, Tom Cooper, Charles Thornton, John Day , Peg Nopoulos , Richard Moxley, Darren Monckton and Gordon Tomaselli.

Dr. John Day and Dr. Charles Thornton then led a robust discussion of the status of current and potential biomarkers, endpoints, and their clinical validation, and what additional work needed to be done to facilitate the clinical trial and drug approval processes. MDF will publish a summary paper on the workshop this Fall.

New Video! Living with DM: The Community Speaks

The conference opened with a new video featuring MDF community members providing moving perspectives of the DM journey and a call to action to help drive Care and a Cure. Click here to watch the 5-minute video.

Keynote Address from Acting US Surgeon General

RADM Boris Lushniak, MD,MPH Acting United States Surgeon General and the ‘Nation’s Doctor,’ opened the conference with an inspirational overview of the importance and specific roles of advocacy, education, research and clinical care in helping improve the quality of life of families living with this disease and driving efforts toward treatments and a cure. He acknowledged the importance of hope and the importance of the current DM journey, and welcomed the community to Washington, DC.

New Breakout Sessions on Vision and Emotional Health

Clinicians and researchers from Stanford University, Washington, DC, University of Iowa and University of Sherbrooke offered outstanding breakout sessions on DM and Emotional Health: Depression and Anxiety, Vision and Eye Health and a best-in-class GI Health session. Visit the MDF Digital Academy to view videos of these sessions, or the 2014 MDF Annual Conference agenda to view and download the PowerPoint presentations.

Expanded Juvenile Onset Adult Ambassadors Track

The JOA Ambassadors track included session break outs by age group, as well as a shared session for JOAs and their caregivers. Generous support from the Cohen Family Trust and AHLIFE (A Healthy Life for Everyone) again provided partial travel scholarships in support of JOA attendance.

Other Conference Programs

An Isis Pharmaceuticals clinical trial update from Dr. Frank Bennett, a preconference adaptive yoga session led by Dr. Ariele Foster, a networking lunch organized by table topics, support group and TeamMDF volunteer meetings, a Kostume Karaoke party and trivia contest at Friday night’s reception and a Twilight Monuments Bus Tour rounded out the action-packed conference weekend.

MDF Announces 2015 MDF Annual Conference Location!

To expedite our advocacy agenda and further build on our community's success in advocating for the recent reauthorization of the MD-CARE Act , MDF will return to Washington, DC, from September 18-19, 2015, for the 2015 MDF Annual Conference. This conference will be held in conjunction with a joint FDA/NIH workshop that we will be hosting. Watch for updates in the coming months. We hope to see you there!

09/26/2014

The Myotonic Dystrophy Family Registry currently has over 1,900 participants, making it one of the largest and most up-to-date myotonic dystrophy (DM) registries in the world. If you’ve been diagnosed with DM1 or DM2, including congenital or juvenile onset, or are the primary caregiver for some who has, and you haven’t already joined the Registry, we need you!

By participating in the Registry you can help researchers from industry and academia identify potential clinical trial participants and research study subjects, and increase understanding of the impact and complexity of this disease.

The Registry is patient-driven, which means you’re in charge of your information. You can opt out of the Registry at any time, and you can also visit the Registry website to review de-identified (anonymous) data and information the same way that registered researchers do. Your individual information is kept completely confidential. Data in patient registries is typically considered out of date and less useful if it is not updated at least every 18 months, so we’ll remind you to log back into the Registry to review and update your survey occasionally.

Click here to go to the Registry website, read and sign the consent form, and get started.

Questions? Contact the Registry Coordinator for more information.

08/20/2014

It's a wrap! A sold-out gathering of over 300 DM families, researchers, medical professionals and vendors from the U.S, Canada and countries as far-flung as Chile came together at the 2012 MDF Annual Conference to learn current strategies for living with DM, gain new information on myotonic dystrophy symptoms, and hear a report on current research advances.

The conference was again a wonderful way for the MDF community to enjoy the camaraderie that the annual conference program promotes. Returning family members reunited with other families that share the DM journey, and met new community members eager to get involved and learn about myotonic dystrophy.

More than a dozen clinical and research professionals presented on topics ranging from DM and the Heart and Family Planning & Genetic Counseling to Occupational Therapy for DM: Strategies for Living and Social Security: Be a Consumer, Not a Victim. A number of conference topics were presented for the first time, and this year's MDF Resource Fair was significantly expanded, to include a broad range of vendors from leading DM research institutions collecting blood samples and data for research studies to vendors demonstrating the latest in orthotic and respiratory assistive devices. It all took place over a sunny, blue-sky weekend at the Hyatt Regency, San Francisco Airport on August 17th and 18th, 2012.

In addition to presentations and Q&A sessions with leading medical providers, this year's conference included a first-ever research update from the pharmaceutical industry, with a presentation from Dr. Robert MacLeod, Vice President of Oncology and Exploratory Discovery at Isis Pharmaceuticals. After an introduction from Dr. John Day of Stanford University that emphasized the importance of continuing to pursue multiple treatment options, Dr. MacLeod discussed current developments in Isis’ research and the company's recent partnership with Biogen Idec.

MDF Board and staff members presented information on new and expanded MDF programs and how MDF community members can get involved, including the Foundation's new initiative, TeamMDF, a grassroots volunteer and advocacy corps that is designed to enhance the support MDF offers to people and families living with DM, generate new tools and resources for the MDF community and create an organized face and voice for myotonic dystrophy at the state and national levels. 

Sunday featured do-it-yourself community meetings and a popular and well-attended chair yoga demonstration. A number of families also took advantage of the lovely Bay area weather to explore San Francisco.

The entire program, including copies of presentations from many of the speakers, can be found by clicking here.

Be sure to mark your calendars for next year's conference! The 2013 MDF Annual Conference will be held November 8th and 9th, 2013 in Houston, Texas. We look forward to seeing you there.

Questions?

Please contact MDF at info@myotonic.org or call 415-800-7777.

08/29/2012

When the Olympic Torch blazes across the UK on its way to the London 2012 Olympics, Margaret Bowler, founder and National Coordinator of the Myotonic Dystrophy Support Group (MDSG), will be one of the 8,000 inspirational individuals honored with escorting the flame along its route. Bearing the Olympic Torch is a great tribute for Margaret, whose own journey through life has been devoted to shining light on the needs of people living with myotonic dystrophy.

A nurse and midwife for 40 years, Margaret directed her energies to the cause of myotonic dystrophy in 1989 when her youngest son and husband were diagnosed with the disease. Frustrated by the lack of scientific research and inadequate knowledge in the medical services, Margaret has spent the last quarter century building the Myotonic Dystrophy Support Group, based in the UK, to help all the families affected by this rare genetic condition.

In response to a nationwide search last summer by the Olympic sponsors for people that had made a “real difference in their communities”, to participate in the Olympic Torch Relay, myotonic dystrophy researcher Dr. Helen Brownlee secretly submitted her name for the honor. The nominating letter cites Margaret’s special qualities that “made the impossible, possible” and reads in part: 

“As National Coordinator of the Myotonic Dystrophy Support Group, she has achieved so much: commissioned the first lay book for families on myotonic dystrophy; sent 187 reassembled wheelchairs to school children with the disease in Bulgaria; raised the awareness of myotonic dystrophy through speaking at national and international conferences and participating in workshops; raised funds to support research on myotonic dystrophy, and most importantly has given families hope by bringing them together with scientists, researchers, clinicians, doctors and other families with myotonic dystrophy.”

To everyone’s delight, word came in early December that Margaret had been chosen from over 60,000 nominees to carry the flame on June 28 in Radcliffe-on-Trent, about 10 miles from where she lives in Nottingham. Dressed in a specially-designed white track suit with gold trimmings, Margaret will carry the flaming, 32-inch torch for approximately two tenths of a mile, while friends, family, church members, and supporters from the MDSG network cheer her along the route.

Says Margaret of the honor, “The amazing thing is that myotonic dystrophy will be publicized, along with my name, and awareness of the condition will be helped tremendously, for sure.” Already she has been interviewed on local radio shows, and stories have appeared in several regional newspapers as well. MDSG is gearing up to take advantage of this fantastic opportunity by printing green and white T-shirts for local supporters to wear in hope that TV cameras will glimpse them in the crowd. Margaret and everyone in the Myotonic Dystrophy community is extremely proud of this great honor and we all wish her the very best for this special day.

05/17/2012