Myotonic Dystrophy Foundation

MINNEAPOLIS, MN (August 13-15, 2010) - More than 200 members of the myotonic dystrophy community met in Minneapolis for a two day conference presented by the Myotonic Dystrophy Foundation in collaboration with the Paul and Sheila Wellstone Muscular Dystrophy Center at the University of Minnesota. Families from 29 states and five countries joined members of the medical and scientific community to discuss the latest updates on research and to get information about diagnosis, treatment and daily living issues. With generous support from our sponsors, Genzyme Therapeutics, Gillette Children's Specialty Healthcare, University of Minnesota, and the University of Rochester Medical Center, the conference was a huge success. The weekend was jam packed with presentations, breakout sessions and opportunities for families to have their questions answered by healthcare professionals and to network with others in the myotonic dystrophy community.

“We all learned so much not only from the lectures but from each other. Thank you for making this all possible, for your dreams and for the hopes we all have for the future.”

Throughout the conference Dr. Richard Lymn fulfilled his duties as Master of Ceremonies with great panache. Dr. Lymn, is a bio-physicist who has dedicated his entire career to muscle research. He continues to foster research on muscular diseases, collaborating with researchers and private groups. Dr. Lymn is a member of the MDF’s Medical and Scientific Advisory Committee.

Dr. John Day, M.D., Ph.D., Professor of Neurology and Pediatrics, Director, Paul and Sheila Wellstone Muscular Dystrophy Center at University of Minnesota spoke at the general sessions on both days and the Saturday afternoon break-out sessions, giving attendees the chance to speak directly with him directly about issues they face on a day-to-day basis. Dr. Day’s presentations were focused on the GI system, the brain, Managing DM2, and next steps in patient registries and drug development therapies.

Dr. Charles Thornton, M.D., Professor of Neurology, Strong Memorial Hospital at the University of Rochester along with Dr. Day and Dr. Auinash Kalsotra, addressed the topic of research updates during the Sunday morning general session. Dr. Thornton also conducted a widely-attended break-out session on “Respiratory Complications and Muscle Weakness in DM1.”

Dr. Auinash Kalsotra, Ph.D., Postdoctoral Fellow, Baylor College of Medicine, Department of Pathology is a current recipient of the MDF’s Fund-A-Fellow grant. Dr. Kalsotra’s research, “Regulatory Mechanisms of CELF Protein in Cardiac Development and Myotonic Dystrophy” aims to identify cellular pathways that are disrupted in myotonic dystrophy type 1 (DM1). He explained his research during a Sunday morning presentation. His presentation was well received by the conference attendees.

In addition to Drs. Day, Thornton and Kalsotra, many other members of the medical and scientific community traveled to Minneapolis to speak on a variety of subjects:

• Dr. Cramer Bornemann, Dr. Jeff Wozniak, Dr. Laura Ranum and Dr. John Day, collectively held a session on the “Brain in DM;”
• Dr. Cynthia Gagnon conducted two sessions on “Health Follow-Up in DM1: Tools for Your Doctor;”
• Dr. Jacinda Sampson and Dr. Chris Boys spoke on the topic of “School Age Child – IEP & Family Support;”
• Dr. Melissa Connolly and Emelia Rogers discussed “Social Services – Advocating for Yourself;”
• Dr. Leslie Krongold, Margaret Bowler of the UK Myotonic Dystrophy Support Group, Claude Bourlier, of AFM in France, Kelly Schuna and Scott Wiebe from the MDA spoke on “Starting a Support Group;”
• Kelly Schuna and Kathryn Ringham held sessions for unaffected “Caregivers;”
• Joline Dalton, genetic counselor at the Paul & Sheila Wellstone Muscular Dystrophy Center at the University of Minnesota spoke on “Genetic Testing & Family Planning.”

On Saturday night, attendees gathered at the nearby Mall of America for a casual evening. Supporting “MDF Benefit Night”, families visited Noodles and Company, Cantina #1, and Kokomo’s Island Café, each of which then made a donation to the MDF based on the total amount of dollars spent at their restaurant. It was a wonderful and relaxing evening and gave many community members a chance to meet in person for the first time.

A highlight of the conference came Sunday morning with a question and answer session with “Global Patient Community Organizations.” The distinguished panel for this Q&A included Claude Bourlier of AFM, France; Margaret Bowler of the UK Myotonic Dystrophy Support Group, Scott Wiebe of the Muscular Dystrophy Association and our own Lisa Harvey, Executive Director of the MDF. The session was very interactive allowing the audience to delve into specific issues and concerns about current and future treatments for myotonic dystrophy.*

Throughout the conference, families took advantage of the free childcare provided by the MDF. We had a wonderful staff of experienced providers, including a registered nurse, who kept the children busy with activities.

The Empower 2010 Family Conference would not have been possible without the assistance of a crew of volunteers who did everything from stuffing bags, to taking notes during sessions, to helping everyone find their way to the right location. It is with a deep sense of gratitude that we thank the following individuals: Margaret Ashton, Carolyn Billing, Sheila Billing, Kathleen Cail, Cassie Carroll, David and Elizabeth Conte, Joline Dalton, Stacey Grimes, Erica Kelly, Steven Kiryakoza, Dr. Leslie Krongold, Karen McConnell, Cameron Naughton, Ashley Peterson, Kelly Schuna, Edibell Stone and Ann Woodbury… you are all the very best!

While attendees were busy learning from the experts and networking with each other, Rick Guidotti of Positive Exposure was busy putting his camera to work. A professional photographer who dedicates his work to capturing the beauty of individuals living with genetic difference, this is the second year Rick has been to an MDF supported conference. Of course, his favorite hang-out was in the childcare room where he captured some of the most precious and precocious conference attendees. Rick’s boundless energy, enthusiasm and love for his work and his models are clear in the photos that he produces.

At the end of the weekend, Lisa Harvey took to the podium encouraged attendees to plan for next year’s Empower 2011 Conference. In our continued support of global research, the MDF will again be a co-sponsor of IDMC, the leading biennial international myotonic dystrophy research conference. With the goal of creating the largest ever patient/research forum, we will hold Empower 2011 in Florida in conjunction with IDMC-8 in December 2011. Check www.myotonic.org, for updates; or, if you haven’t already registered with us, follow this link and sign up for a Family Empowerment Pack and we will keep you up-to-date on the Empower 2011 conference details.

In closing, we were please to be able to offer this conference, including the conference venue, meals and conference materials, free of charge to families living with myotonic dystrophy. Donations during the conference totaled more than $10,000 which will be used to continue funding educational, research, and advocacy programs, including future conferences. If you would like to help the MDF to continue its work, please click here.

*Please note that additional information from these and other sessions will be available shortly on the MDF website, www.myotonic.org.