2022 MDF Annual Conference
Paradise Point, San Diego, CA
September 9th & 10th
The DM Community Reunites at the 2022 MDF Annual Conference
The 2022 MDF Annual Conference - sometimes called the “family reunion” - took place from Friday, September 9th through Saturday, September 10th, 2022, at Paradise Point in San Diego, CA. With over 625 registrants, and hundreds of participants in-person & online - joining from 39 US States and 14 countries - we are thrilled that so many of our incredible community members and professionals could once again unite to advance the MDF mission of Community, Care, and a Cure!
MDF offers a special thank you to Avidity Biosciences for hosting two laboratory tours the Thursday before the Conference for 40 members of the DM Community to learn more about their drug development process and progress towards a treatment for DM1.
This year's program included 23 educational sessions exploring research, drug development, symptom management, and other critical topics for DM families, plus opportunities for formal and informal networking and fun. Over 184 Conference first-timers from across the country attended our First Timers’ Tea to connect with other families new to the community, board members and support group facilitators. Attendee feedback on the impact of gathering in person was overwhelmingly positive, and further solidifies the MDF Annual Conference as the crown jewel of MDF events!
Congratulations to Our Award & Raffle Winners
MDF is thrilled to congratulate and honor our three award winners for their uncontested commitment and service to the myotonic dystrophy community:
- Emily Jones
4th Annual Kayla Vittek Memorial Award for Outstanding Community Advocate, presented by Lisa Harvey-Duren
- Luke Bolt
Above & Beyond Award, presented by MDF's CEO, Tanya Stevenson, EdD, MPH
- Tetsuo "Tee" Ashizawa, MD
Lifetime Achievement Award, presented by Larry Lord & Darren Monckton, PhD
Thank you to everyone who participated in the 2022 MDF Annual Conference Raffle! Your support helps funds our critically important work on behalf of individuals and families living with myotonic dystrophy. Congratulations to the raffle winners!
- Naomi Scarberrry, Amazon Gift Card
- Mary Jane Toolson, Amazon Gift Card
- Richard Weston, iPad
- Merrily Miller, Gold & Tourmaline Necklace
- Tammy Reid, Mammoth Lakes Vacation
DM Research Poster Abstracts
Traditional poster sessions combine a visual representation of the findings of a paper/study – the poster - with the opportunity for individualized, informal discussion of the presenter’s work. MDF was thrilled to make this opportunity available at the 2022 MDF Annual Conference in a hybrid format, where attendees could view posters in-person and virtually. Click here to view the 2022 MDF Annual Conference DM Research Poster Abstracts.
Watch the Recorded Sessions
MDF is actively working to make recorded sessions publicly available on the Digital Academy as soon as possible! Click on the hyperlinked titles below to access each sessions recording.
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.
Sessions not listed below were not recorded by request or in order to protect the privacy of the live attendees.
The Conference incorporated many traditional events and session such as updates on drug development, highlights of recent successes and future goals of the foundation, and inspiring stories from the community.
- Welcome & State of the Foundation
MDF leadership and Board of Directors were eager to share exciting updates about the state of Foundation, International Myotonic Dystrophy Awareness Day, and the MDF strategic plan.
- Stories of Inspiration from the DM Community
A celebration of the DM community, including the 4th Annual Kayla Vittek Memorial Award for Outstanding Community Advocate presented by Lisa Harvey-Duren. Also awarded were the Above & Beyond Award and Lifetime Achievement Award.
- Industry Updates Part 1
Representatives from biotech and pharma companies provided updates on their drug development efforts in the DM field. Part 1 companies include: Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, GrittGene, and AMO Pharma Ltd.
- Industry Updates Part 2
Representatives from biotech and pharma companies provided updates on their drug development efforts in the DM field. Part 2 companies include: PepGen, Juvena Therapeutics, Entrada Therapeutics, NeuBase Therapeutics, and ARTHEx Biotech.
- First Timers' Tea
A networking event for those who have not attended a Conference before to orient you to the Conference, MDF, and the DM community.
- Disability Rights & Resources
Presented by Christopher Knauf, Esq.
Many individuals living with DM – types 1 and 2 – and their families may find themselves in situations where they need to know their rights, especially rights related to employment, education, public accommodation, genetic counseling, and healthcare. This workshop provided an overview of key laws such as the ADA, IDEA, ACA, GINA, which may protect you or your family. It will cover some common situations when these laws can be used and address processes for activating those rights, including when to consider engaging a lawyer or other advocate. Learn, too, about organizations, agencies, and other resources to help empower yourself and your family.
- The Wide World of Research & Securing Funding to Move Us Closer to A Cure!
Jacinda Sampson, MD of Stanford University and Kevin Brennan of Bluebird Strategies
Learn about biobanks, natural history studies, clinical trials, registries, and other types of research, the role that they play in drug development, and how DM1 and DM2 families can participate right now. Hear how MDF has and continues to facilitate research towards a cure, and discover ways that you can help increase federal funding for DM research right now!
DM Type 1
This track was designed for individuals and families living with myotonic dystrophy type 1 (DM1).
- Understanding DM1: Disease Development & Symptom Management
Chamindra Konersman, MD of UC San Diego Health
Presented by Chamindra Konersman, MD. Learn from a leading expert about the causes and genetics underlying DM1, how and when in life it can affect different systems of the body, tips to help self-manage symptoms, and best practices for working with a care team to ensure the best quality of life. Learn, too, about the latest research in DM1 and how you can get involved.
- DM1 Community Panel
Mindy Kim, Bill Nuttall, Jeannine DeSoi, and Dean Sage
Hear from a panel of individuals with DM1 about how they live their best life.
DM Type 2
This track was designed for individuals and families living with myotonic dystrophy type 2 (DM2).
- Understanding DM2: Disease Development & Symptom Management
Thurman Wheeler, MD of Massachusetts General Hospital
Learn from a leading expert about the causes and genetics underlying DM2, how and when in life it can affect different systems of the body, and how best to self-manage and work with a care team to ensure the best quality of life. Learn, too, about the latest research in DM2 and how you can get involved.
- DM2 Community Panel
Tom McPeek, Lorrie Gallagher, E. Kahlifa, and Elyse Posner
Hear from a panel of individuals with DM2 about how they live their best life.
- DM2: The Brain & the Muscle
Araya Puwanant, MD of Wake Forest University
This session will start with a short review of the CNS manifestations in DM2 followed by a discussion of pilot data in everyday terms from a recent DM2 brain imaging and cognitive study. It will also include a discussion of how brain abnormalities could potentially affect motor function.
Caregivers & Self-care
This track was designed for family members, caregivers, those who are their own caregiver; anyone living with any type of DM.
- Making Daily Life Easier with an Occupational Therapist
Samar Muslemani, OT, MSc of the University of Sherbrooke
A leading clinician and researcher in occupational therapy (OT) defines this form of healthcare and advise on how it can be used by individuals with DM1 and DM2 and their families. She also discusses the ways that adaptation, task alteration, devices, or environmental changes can help individuals with DM, both children and adults, make their daily life easier. She specifically addresses common issues and solutions in different activities such as moving, toileting, and eating for adults and children.
- Physical Therapy, Mobility & Adaptive Sports
Melissa McIntyre, DPT of the University of Utah Health
Join a leading physical therapist whose clinical care and research focus centers on individuals with neuromuscular disorders. In this session, she discusses the basics of physical therapy (PT) and what it can do for DM populations – both type 1 and type 2, adult and pediatric. Additionally, this session reviews adaptive sports and novel adaptive equipment.
- Unaffected Female Caregivers Community Panel
Suzanne Perkins, PhD, Kim McPeek, Emily Jones, and Julie LeBoeuf
Hear from a panel of spouses and other caregivers about how they live their best life.
This track is specifically designed for clinicians, researchers, industry, and other DM professionals.
- Considerations & Open Discussions of Exercise Impact on Clinical Trials & Everyday Health
Thomas A. Cooper, MD, Mark Tarnopolsky, MD, PhD, FRCP(C), Elise Duchesne, PhD, Andrew Berglund, PhD, and Tina Duong MPT, PhD
The Professional Track begins with a session on DM and exercise. Thomas A. Cooper, MD, presents, Endurance exercise leads to beneficial molecular and physiological effects in the HSALR mouse model of myotonic dystrophy type 1. Mark Tarnopolsky, MD, PhD, FRCP(C) presents, Exercise and nutritional strategies for myotonic dystrophy. After a short break, Elise Duchesne, PhD, presents, Strength training effectively alleviates skeletal muscle impairments in myotonic dystrophy type 1. Andrew Berglund, PhD., presents, the Analysis of individual transcriptomic response to strength training for myotonic dystrophy type 1 patients reveals rescue at the molecular level. The session will end with a presentation by Tina Duong MPT, PhD.
- Future Directions of DM Research - Part 1
Andrew Berglund, PhD and John Lueck, PhD
First in our lineup of presentations on future directions in DM research, John Lueck, PhD, presents, Generation of Bi-channelopathy DM Mice and Drug Repurposing. Then, Dr. Berglund returns to the podium to present on screening and natural products.
- Future Directions of DM Research - Part 2
Laura Ranum, PhD and Eric Wang, PhD
Opening the afternoon sessions on Future Directions of DM Research is Laura Ranum, PhD, presenting, A BAC transgenic mouse model for DM2. Afterward, Eric Wang, PhD, will ask the question: is gene therapy in the cards for myotonic dystrophy?
- Research Dataset Presentations
Suzanne McDermott, PhD, Nicholas Johnson, MD, and Jeffrey Statland, MD
Suzanne McDermott, PhD, presents, Groups of Signs and Symptoms in adult-onset myotonic dystrophy type 1. Nicholas Johnson, MD, and Jeffrey Statland, MD, present, The Myotonic Dystrophy Clinical Research Network (DMCRN): observational studies as the foundation for clinical trials.