2023 MDF Annual Conference

Renaissance Washington DC Downtown Hotel
September 7th, 8th & 9th
 

MDF Returns to Washington DC for Our Largest Conference Ever

Thank you so much for joining us in Washington DC, and online, for the 2023 MDF Annual Conference! The 2023 MDF Annual Conference - sometimes called the “DM family reunion” – kicked off with MDF Advocacy Day on Capitol Hill on Thursday, September 7th, and continued with our educational and networking sessions through Saturday, September 9th, 2023, at the Renaissance Washington, DC Downtown Hotel.

With over 600 in-person and virtual attendees from 18 countries - we are thrilled that so many could join us to make lifelong memories and experiences! We hope you enjoyed this year's sessions exploring research, drug development, symptom management, and our opportunities for formal and informal networking, wellness, and fun.

Click here to view all the photos from the 2023 MDF Annual Conference on Facebook!

Congratulations to Our Community Award Recipients!

MDF is proud to congratulate and honor our four Community Award recipients for their uncontested commitment and service to the myotonic dystrophy community:

• Margaret Bowler
  Lifetime Achievement Award
   
• Rebecca Coplin
  5th Annual Kayla Vittek Memorial Award for Outstanding Community Advocate
   
• Ann Woodbury
  MDF Support Group Facilitator Warrior Award
   
• Kristen McClintock
  MDF Support Group Facilitator Warrior Award

MDF Advocacy Day on Capitol Hill

On Thursday, MDF hosted MDF Advocacy Day on Capitol Hill to meet with Members of Congress and their staff to advocate for DM research funding and awareness. (Special thanks to our Lead Conference Sponsor, Avidity Biosciences, for sponsoring our Community Breakfast & Advocate Training!) After reviewing everything you need to know for a successful congressional meeting, attendees boarded a shuttle bus to meet their representatives on Capitol Hill.

Over 130 myotonic dystrophy advocates visited over 100 congressional offices on behalf of the DM Community! We are grateful to all who joined us for a special MDF Congressional Briefing that included presentations from Senator Klobuchar, the Department of Defense, the National Institutes of Health, Avidity Biosciences, and members of MDF’s National Advocacy Committee.

Click here to read more about MDF Advocacy Day on Capitol Hill.

The day concluded with a Welcome Reception & Advocacy Awards Presentation, where we recognized Senators Tim Kaine (D-VA) and Cynthia Lummis (R-WY) and their staff for their friendship and support of the myotonic dystrophy community.

Click here to learn more about advocating for the DM Community!

Magic & Mystery at the MDF Annual Conference

Throughout the Conference, you could feel the magic in the air! At our Friday Dinner, the spectacular magician & mentalist, David Gerard, put on a legendary performance that left even some of our most skeptical scientists and board members scratching their heads!

Our Saturday night Dinner & Dance Party kept the surprises coming! Thank you to everyone who participated in our Mystery Box Challenge! Your support helps funds our critically important work on behalf of individuals and families living with myotonic dystrophy. Congratulations to our three winners!

• Ginny Morris, a trip to the 2023 MDF Gala!
• Ritika Puri, a brand new iPad!
• Angie Wiggans, a $250 Amazon Gift Card!

Congratulations Iberia & Jackson! Before our community began breaking out their signature dance moves, an incredibly brave young man named Jackson Cooley made his way to the stage for a surprise marriage proposal to Iberia Dunning! He shared how the two had met and become friends through the JOA track of the MDF Annual Conference 5 years ago and had since fallen in love! Congratulations to the future bride and groom!

Improving the Conference Experience

In response to community feedback, we were delighted to offer new programs designed to better connect our diverse community and improve their Conference experience.

Networking Buddies: Connecting the DM Community and Research Professionals. An exciting and new way to get to know one another, the Conference Networking Buddies system paired DM Professionals with people living with DM and their caregivers. In addition be seated together at Friday's Lunch, Conference networking buddies continued to get to know each other during snack breaks, cocktail hours, and poster sessions.

The Wellness Room, brought to you by Harmony Biosciences. New to this year’s conference is the Wellness Room. Throughout Friday & Saturday, the Wellness Room welcomed attendees to take advantage of a delightful array of activities designed to relax, rejuvenate, restore:

• Quiet Spaces: Step away from the conference buzz to find tranquility in our designated quiet space filled with comfortable lounge furniture
• Movement & Mindfulness Moments: Join our experienced instructors specializing in working with DM and/or other neuromuscular disease communities for invigorating movement moments and soothing mindfulness exercises
• Fireside Chats: Engage with leading experts in insightful 45-minute-long small group discussions covering a wide range of topics.

Our Program & Recorded Sessions

The 2023 MDF Annual Conference hosted a wide range of topics of interest for our Community presented by leading experts in DM.

Click here to download a PDF of the MDF Annual Conference Program Book!

MDF is actively working to make recorded sessions publicly available on the Digital Academy!

The MDF Digital Academy: Re-watch and share your favorite presentations and learn more about myotonic dystrophy with our extensive catalog of video resources. Watch previous conference sessions, webinars, and technical explanations on DM. Find hope and inspiration from community members sharing their stories and learn about the impact of the Foundation. Click here to start exploring the MDF Digital Academy.

Please note, sessions marked with an * were not recorded by request or to protect the privacy of live attendees.

General Sessions

First Timers’ Tea*

The First Timers’ Tea is an informational and networking event for those who have never before attended an MDF Conference. This session will be led by MDF and community leaders, and is designed to orient families to the Conference, to MDF, and to the DM Community.  

Welcome & State of the Foundation

MDF leadership and Board of Directors will share exiting updates about the Foundation, International Myotonic Dystrophy Awareness Day, and discuss progress in the field of DM. 

DM Drug Development and Approval Considerations with the FDA

Nicholas Johnson, MD, MSci., FAAN, Virginia Commonwealth University
Michelle Campbell, PhD, Federal Drug Administration
Ami K. Mankodi, MD, Federal Drug Administration 

Gain insight and understanding on the important role the FDA plays in the drug approval process through a presentation from the FDA. This informative presentation will be followed by a panel discussion with the FDA, a DM expert clinician, and members of the DM Community. This session provides an opportunity for our community and clinicians to share the serious medical challenges facing our community with the FDA.  

Industry Updates

With an opening statement from Dr. Andy Berglund, MDF Scientific Advisory Committee member, representatives from biotechnology and pharmaceutical companies will provide updates on their drug development efforts in the DM field.

Industry Updates Day 1: Avidity Biosciences, Juvena Therapeutics, Sanofi & ARTHEx Biotech.

Industry Updates Day 2: Dyne Therapeutics, PepGen Inc., Harmony Biosciences, Rgenta Therapeutics & AMO Pharma.

Stories of Inspiration from the DM Community

Start the day off with a celebration of the DM community, as community leaders step onto the stage to receive special awards, including the 5th Annual Kayla Vittek Memorial Award for Outstanding Community Advocate, the Above & Beyond Award, and Lifetime Achievement Award.  

Exhibitor and Research Poster Showcase*

Network and connect with poster authors conducting DM research, industry partners, and others working in the DM space. Click here to download the 2023 Research Poster Abstracts.  

DM Type 1

DM1: Disease Development and Symptom Management

Johanna Hamel, MD, University of Rochester Medical Center 

This session is ideal for those new to the DM1 community and those seeking a refresher. Learn from a leading expert about the causes and genetics underlying DM1, how and when in life it can affect different symptoms of the body, tips for self-management of symptoms, and how to best work with your clinical care team to ensure the best quality of life.  

DM1: Impact of Cognitive Impairments on Daily Living

Samar Muslemani, MOT., MSc. PhD, Universite de Sherbrooke

Issues related to cognitive impairments are sometimes misunderstood, especially when considering how they may relate to independence and activities of daily living (ADLs). These misunderstandings can sometimes create conflicts or prejudice. Learn from a leading expert about how cognitive impairments influence the ability to accomplish ADLs and social roles. And how occupational therapists and other healthcare professionals can help with ADL difficulties related to cognitive impairments.

DM1 Networking*

Networking sessions at the conference provide safe and welcoming environments to connect and engage with your peers through discussions led by MDF Support Group Facilitators.

Managing Sleepiness & Sleep Disturbances in DM

John Day, MD, PhD, Stanford University 

This session will cover sleepiness and other sleep disturbance considerations for both DM1 and DM2. Learn from a leading expert about sleepiness, and other sleep disturbances related to DM1 and DM2, best practices for care, monitoring, and self-management. Learn also about the latest research and how you can get involved!

DM1 Community Discussion Panel: Life Hacks*

Community Discussion Panels at the conference are sessions lead by MDF volunteer community leaders. Each community has a dedicated discussion panel and topics chosen by community leaders.

DM1 Stump the Doctor*

Ericka Greene, MD, Houston Methodist

Stump the Doctor Sessions at the conference are an opportunity for community members to meet a leading expert in myotonic dystrophy and ask their most persistent, challenging questions. Can you stump the doctor?

DM Type 2

DM2: Disease Development and Symptom Management*

Chad Heatwole, MD, MS, University of Rochester Medical Center 

This session is ideal for anyone within the DM2 community. Learn from a leading expert about the causes and genetics underlying DM2, how and when in life it can affect different systems in the body, monitoring, and how best to self-manage and work with your care team to ensure the best quality of life. Learn, too, about the latest research in DM2 and how you can get involved. 

DM2 Networking & Social Hour*

Networking sessions at the conference provide safe and welcoming environments to connect and engage with your peers through discussions led by MDF Support Group Facilitators.

Managing Sleepiness and Other Sleep Disturbances

John Day, MD, PhD, Stanford University 

This session will cover sleepiness and other sleep disturbance considerations for both DM1 and DM2. Learn from a leading expert about sleepiness, and other sleep disturbances related to DM1 and DM2, best practices for care, monitoring, and self-management. Learn also about the latest research and how you can get involved!

DM2 Community Discussion Panel*

Community Discussion Panels at the conference are sessions lead by MDF volunteer community leaders. Each community has a dedicated discussion panel and topics chosen by community leaders.

Gastrointestinal Considerations in DM2

Irene Sonu, MD, Stanford University

This session explores the ways in which DM2 affects the GI system, treatment and management strategies aimed at alleviating GI issues, and improving quality of life.

DM2 Stump the Doctor*

Araya Puwanant, MD, Wake Forest University

Stump the Doctor Sessions at the conference are an opportunity for community members to meet a leading expert in myotonic dystrophy and ask their most persistent, challenging questions. Can you stump the doctor?

DM2: Cardiac Considerations

Pradeep P.A. Mammen, MD, FACC, FAHA, FHFSA, University of Kansas Medical Center 

Learn from a leading expert about the effect of DM2 on the heart. 

CDM Families

CDM: Communication Considerations in Congenital Myotonic Dystrophy Workshop*

Kiera Berggren, MA/CCC-SLP, MS, Virginia Commonwealth University 
Lauren Brand, MS, CCC-SLP, Lauren Brand Speech 

This working session begins with a short presentation on communication concerns in CDM, followed by a structured discussion on the considerations for parents and caregivers regarding communication in children with CDM.

CDM Family Networking & Social Hour*

Networking sessions at the conference provide safe and welcoming environments to connect and engage with your peers through discussions led by MDF Support Group Facilitators.

CDM: What to do Before Your Child Turns 18*

Join this panel of experts as they share important considerations to consider before your child with CDM turns 18. 

Caregivers

Caregiver Networking & Social Hour*

Networking sessions at the conference provide safe and welcoming environments to connect and engage with your peers through discussions led by MDF Support Group Facilitators.

Unaffected Male Caregivers Community Discussion Panel*

Community Discussion Panels at the conference are sessions lead by MDF volunteer community leaders. Each community has a dedicated discussion panel and topics chosen by community leaders.

Unaffected Female Caregivers Community Discussion Panel*

Community Discussion Panels at the conference are sessions lead by MDF volunteer community leaders. Each community has a dedicated discussion panel and topics chosen by community leaders.

Topic Based Breakouts

Social Security Administration Demystifies the Benefits Process

Diana Varela, Public Affairs Specialist, Social Security Administration 
Joyce Lee, Medical Relations Officer, Department on Disability Services

Gain a better understanding of the role of the Social Security Administration (SSA) programs, benefits, and services available to the DM community in the US. Leaders from the SSA will join this panel to help demystify the application, denial, and appeal process.  

Stump the Doctor: Mobility*

Tina Duong, MPT, PhD, Stanford
Katy Eichinger, PhD, DPT, University of Rochester Medical Center

Stump the Doctor Sessions at the conference are an opportunity for community members to meet a leading expert in myotonic dystrophy and ask their most persistent, challenging questions. Can you stump the doctor?

New Horizons in the DM Field

John Day, MD, PhD, Stanford
Charles Thornton, MD, University of Rochester Medical Center 

Hear about the significant progress in the DM field from leading experts during this panel discussion. Learn about the impact the community can have on continuing the progress toward treatments and a cure.  

Mental Health & Myotonic Dystrophy: Answering Your Questions*

Melissa Dixon, PhD, University of Utah 
Benjamin Gallais, PhD, Universite de Sherbrooke 
Ruth Sheldon, MPH, MSW, Myotonic Dystrophy Foundation

Join this panel of experts as they share insight and engage in dialogue with the audience to answer questions about mental health, and the new MDF Mental Health Handbook. Visit the Mental Health Handbook poster in the Poster Showcase and add your anonymous question to be asked during the session.

Juvenile-onset Adults (JOA)

MDF designed the juvenile-onset adults (JOA) sessions with community input from JOAs and these sessions were only open to the JOA community. JOA is often defined as someone with DM1 whose symptoms manifested in childhood or early adulthood, usually before age 21. These sessions were designed to provide networking, connection and resource opportunities for this unique community. In addition to the sessions outlined below, the JOA lounge hosted the following activities  during “open lounge” times: 

• Card and board games (Uno, Balloon Volleyball, Mafia, etc.) 
• Group Games (Selfie Challenges, Order Challenges) 
• Karaoke 

Getting to Know You & Ice-Breaker Games*

Let’s get to know each other with some awesome team building games. We will work together to list the group rules for the JOA Lounge. The Lounge will be open to JOA members throughout the conference so plan to stop by and chill out whenever you need to relax!

Building Social Connections with Others*

Melissa Dixon, PhD, University of Utah 

This session will focus on working with the group on how to build social ties to promote meaningful connections with friends and significant others. This session is open only to JOA community members.  

Let’s Talk About Feelings*

Melissa Dixon, PhD, University of Utah 

Back by popular demand, this session will focus on how feelings affect our bodies and minds. This will include an interactive discussion about strategies for learning to manage overwhelming emotions.  

JOA Stump the Doctor*

Jacinda Sampson, MD, PhD, Stanford 

Stump the Doctor Sessions at the conference are an opportunity for community members to meet a leading expert in myotonic dystrophy and ask their most persistent, challenging questions. Can you stump the doctor?

Wellness Room - Sponsored by Harmony Biosciences

New to this year’s conference is the Wellness Room. This room was open throughout both days of the conference and offered an opportunity for attendees to take a break from sessions, noise, crowds, etc. 

Movement

All movement sessions in the Wellness Room are taught by instructors who have experience working with DM and/or other neuromuscular disease communities. 

Friday Morning & Afternoon Chair Yoga*

Ellen Shapiro, C-IAYT 

These hour-long movement sessions are for all levels and experience. 

Focus on Breathing Techniques*

Lorna Jarret, D.H.Sc. 

This 45-minute session provides an overview on mindful breathing techniques. 

Saturday Morning & Afternoon Pilates, Gentle & Chair*

Lorna Jarret, D.H.Sc. 

These hour-long movement sessions are for all levels and experiences. 

Fireside Chats

Fireside chats were 45-minute-long discussions on a variety of topics with leading experts. These sessions are inclusive and welcoming and intended to provide a supportive environment for all to share their experiences.

Managing Activities of Daily Living*

Samar Muslemani, MOT., MSc. PhD, Universite de Sherbrooke

Myotonic Dystrophy can impact all aspects of your life. This session focuses on the tools and resources available to live with DM. Whether you attended Samar’s session on this topic earlier in the day, or missed it, this fireside chat is an excellent opportunity for everyone to get your questions answered and hear from fellow attendees.  

Effective & Empathic Communication*

Dedee Caplin, PhD, University of Utah 

This session provides an excellent opportunity to explore ways of communicating with your workplace, healthcare team, friends, and family. Learn how to share your experiences with people who don’t understand DM or its impact. 

Clinical Trials & Studies*

Nicholas Johnson, MD, MSci, FAAN, Virginia Commonwealth University 

Progress in DM research is full steam ahead, and there is a lot of hope for new treatments and a cure. Dr. Nick Johnson brought his expertise to a small group discussion where attendees asked questions about participating in clinical trials, natural history studies and more!

How to Raise Awareness for DM *

Kevin Brennan, Bluebird Strategies 

Learn how you and your family can make a big impact in raising awareness for DM within your communities and local and national governments. Whether you joined MDF’s Advocacy Day this year, or you’re looking to help make change in DM, this session is a great opportunity to learn more about how you can get involved. 

DM Professionals

Sessions specifically designed for clinicians, researchers, and industry experts. Session topics were developed based on direct feedback from professionals. Click here to view the DM Professional Session Abstracts.

Click here to download the 2023 DM Research Poster Abstracts.

Presentations listed in order of appearance:

Involvement of Aberrant Splicing of the NFIX Transcription Factor in Transcriptome Changes in Muscles of DM Patients*

Krzysztof Sobczak, PhD, Instytut Biologii Molekularnej i Biotechnologii, Adam Mickiewicz University 

Toxic RNA Selective Screening to Identify New Drugs, Drug Targets and Genetic Modifiers for Myotonic Dystrophy

Kalak Reddy, PhD, State University of New York at Albany’s RNA Institute 

Use of Human Pluripotent Stem Cells for Deciphering Myotonic Dystrophy Type 1 

Cécile Martinat, PhD, I-Stem - Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques 

Timing is Everything: Understanding Sleep Dysregulation in Myotonic Dystrophy*

Belinda Pinto, PhD, University of Florida at Gainesville

Age-related Corneal Disease Mediated by Expanded CUG Repeat RNA

Vinod Mootha, MD, University of Texas Southwestern Medical Center 

Genetic Modifiers of Huntington Disease: Biological Insights and Therapeutic Opportunities

Darren Monckton, PhD, University of Glasgow 

Brain Disease Mechanisms in Myotonic Dystrophy and Why Neurons Aren't the Whole Story 

Mario Gomes-Pereira, PhD, Sorbonne Université, Inserm, Centre de Recherche en Myologie, Paris, France 

Accelerating Knowledge: Research Poster Lightning Round

Moderated by: Tom Cooper, MD, Baylor College of Medicine

Johanna Hamel, MD, University of Rochester Medical Center

Janel Peterson, Baylor College of Medicine

Claudia Lennon, The RNA Institute, State University of New York at Albany 

Laura Girard-Côté, Université du Québec à Chicoutimi (UQAC), Canada

Preeti Kumari, PhD, Massachusetts General Hospital

Emily Davey, University of Florida at Gainesville

Curtis Nutter, PhD, University of Florida at Gainesville

Subodh Mishra, PhD, The RNA Institute, State University of New York at Albany

Lily Cisco, University of Rochester

Disease Severity and Progression in Myotonic Dystrophy Type 2

Johanna Hamel, MD, University of Rochester Medical Center 

Insights Into muscle Pathology: Imaging Analysis and Clinical Endpoints in Myotonic Dystrophy Type 2

Araya Puwanant, MD, MS, Wake Forest University School of Medicine 

Generation and Characterization of a DM2 BAC Mouse Model*

Avery Engelbrecht, Graduate AST-R, University of Florida at Gainesville

Thank You to Our Conference Sponsors

Special thanks to our Sponsors for helping make this event possible. An additional thank you to Avidity Biosciences for sponsoring Storytelling Impact Training for our community panelists and advocates.

For questions on corporate sponsorship opportunities, please contact MDF at development@myotonic.org.