Lots of Firsts
The 2018 MDF Annual Conference is in the rear-view mirror, and it was one for the books. We had over 430 registrants - our largest gathering of family members, academic and industry professionals ever. Located at the Gaylord Opryland Resort & Convention Center, the meeting was our first conference hosted in the South since 2011.
First Timers’ Mentor Program
The conference also featured other firsts, including our largest number of first-time attendees (over 140), and the most representatives from industry at an MDF conference to date. MDF also launched a First-Timers Mentor Program at this year’s conference. Board members and support group facilitators were assigned to first-time attendees, walking them through tips for conference navigation at the First Timers’ Tea, and helping them out whenever needed throughout the conference.
Consensus-based Care Recommendations for Adults with Myotonic Dystrophy Type 1
We had our first session featuring a series of skits, designed to help community members understand the importance of the Consensus-based Care Recommendations for Adults with Myotonic Dystrophy Type 1, which were published online on September 13, the day before the conference kicked off. In the skits, clinical professionals acted as patients and caregivers, and family members performed as doctors in living room and doctors’ office settings to help role play how to introduce and share the Recommendations with your care providers.
These are the only comprehensive, published clinical care recommendations for this disease, because an evidence-based guideline to direct clinical care is not available, and won’t be in a substantive way until much more evidence and data are collected via rigorous studies. More than 66 clinicians experienced in the care of DM1 patients in Europe, Canada, the UK and the US worked collaboratively for well over a year to develop consensus on recommendations for care in the clinical setting. Everyone at the Conference received the Quick Reference Guide (QRG) version of the Consensus-based Care Recommendations for Adults with Myotonic Dystrophy Type 1 to share with their doctors.
Watch for a comprehensive overview of the Recommendations when the print version of the article in Neurology Clinical Products is released for more information on these recommendations and why they are critical for improving international myotonic dystrophy care.
New! The MDF Clinic Visit Planner
MDF also introduced the MDF Clinic Visit Planner during the skits, handing out laminated Planners with erasable pens to enable families to discuss upcoming clinic visits and jot down important questions and information to help ensure that it was shared at the appointment. The Planners were a big hit, and many family members have contacted the Foundation to request additional copies. The Planner is available on the MDF website or by request to MDF.
Robust Professionals Track Reported on the State of Research and Drug Development
The closed Professionals Track ran all day Friday, and included more than 110 academic, industry, investment and federal agency professionals discussing current research updates on actual disease prevalence, a burden of disease study produced with Mayo Clinic and Optum Labs, updates on the status of biomarker and endpoint development, current developments in research on myotonic dystrophy type 2, congenital myotonic dystrophy, and much more. MDF and the Wyck Foundation funded most of these studies and research efforts.
Research Updates and Strategies for Symptom Management
The Conference also featured community sessions focused on managing disease symptoms and maintaining quality of life, including exercise to maintain strength and mobility, tips for understanding and managing GI and respiratory health, and a first-ever patient report out on the use of cannabis (marijuana) and cannabinoids to manage disease symptoms. Access all videos of taped sessions, and presentations here.
Understanding Disease Changes and Progression to Drive Therapy Development
MDF also continued the community panel session format at this year’s conference, building on the popularity of the Patient-Focused Drug Development (PFDD) meeting on myotonic dystrophy that MDF held with the Food and Drug Administration (FDA) in Washington, DC in 2016. The PFDD meeting, the first externally-led meeting approved by the FDA, included over 250 participants and 8 community panelists who helped regulators responsible for reviewing potential drugs to understand what is most burdensome for patients living with DM, and what they seek from eventual therapies.
The 2018 version of this meeting format focused on the issue of disease changes over time and progression, a complicated topic in myotonic dystrophy that needs to be better understood to facilitate the design of therapies and clinical trials to test them. The reports from family members on what had triggered changes in their myotonic dystrophy symptoms, what they had tried to manage these changes, and what they would consider a meaningful benefit from a therapy that targeted progression were fascinating. There were many common threads: pregnancy, the flu or an illness and falls were often cited as progression triggers, and neither panelists or audience members who participated in the open mic discussion could identify successful strategies for preventing or slowing progression. MDF is producing a formal report on this session, which can be watched here. The report should be published and available later this year.
Pizza Parties, Western Line Dancing, the Grand Old Opry and an Art Auction
There were many other highlights, including a research update lunch for DM2 family members, an outing to the Grand Old Opry, pizza party and DJ night for the juvenile-onset adults (JOAs) who attended, a silent auction of beautiful original watercolors and drawings by late community member and artist Lee Baker, an adaptive line dancing lesson during the western-themed closing night dinner and dance party, and more. All in all, one for the books!
2019 MDF Annual Conference – September 13-14, 2019 in Philadelphia, PA!
MDF staff are now reviewing the results of the post-conference survey and other information, and talking with community members and professionals to start planning the 2019 MDF Annual Conference. Mark your calendars for Friday September 13 and Saturday September 14, 2019 at the Loews Philadelphia Hotel in Philadelphia. We hope to see you there!