2019 MDF Annual Conference Agenda

The annual Drug Development Roundtable brings together leaders from academia, biotechnology and pharmaceutical industries to discuss opportunities, barriers and pre-competitive space collaborations to facilitate the pathway to drugs and biologics for DM. As part of the 2018 Roundtable, Dr. Mankodi will discuss results from the Multicenter Observational Study of Myotonic Dystrophy Type 1, Dr. Johnson will present an update on the Trial Readiness and Endpoint Assessment in cDM1 Study, and Dr. Gagnon will be sharing results from a 9-Year Longitudinal Study in DM1 Patients.

Check in and receive your free conference bag! (Includes bracelets, a pen, a program, and networking cards)

Dr. Johnson will provide results of the MDF and Wyck Foundation-funded population-based prevalence study that used 70,000 randomly selected, de-identified newborn blood spots and high-throughput screening to examine the genetic prevalence of DM1 and DM2 in the general population.

A closed meeting for MDF Support Group Facilitators, hosted by the MDF Program Director. Brunch will be provided.

The number of current studies in DM2 basic and translational science has been increasing. Presenters will focus on new preclinical research findings and translational work being conducted in DM2.

A welcome session for first-time attendees. Learn tips for having a successful conference experience from MDF Board members, support group facilitators and experienced attendees and pick up a session cheat sheet. Light snacks and beverages will be served.

Dr. Lott will present the results of his MDF and Wyck Foundation-funded study exploring the development of magnetic resonance imaging as an endpoint in myotonic dystrophy type 1; Dr. Nopoulos will provide an overview of the current NINDS-funded U-IA MRI study on brain structure and function in adults with a family history of myotonic dystrophy type 1; Dr. Thornton will present an update on the development of a muscle splicing biomarker for DM; Dr. Wheeler will provide a report on his MDF and Wyck Foundation-funded study of serum biomarkers to help evaluate disease severity and the efficacy of potential DM1 and DM2 therapies.

The opening session will include a welcome and Care and Cure progress update from MDF CEO Molly White.

Gut Instincts: GI Symptom Management
Leila Neshatian, MD, MSc, Stanford University. Dr. Neshatian will provide an overview of how DM can affect the GI tract, from swallowing issues to gut and processing concerns, and how affected individuals can manage these symptoms.

A program designed specifically for juvenile-onset adults, facilitated by MDF community members Barry Cohen, Ph.D and Diane Bade, RN, CAVS.

Visit the Research & Vendor Showcase in the Governor's Lobby to talk to researchers about current studies, and talk with industry professionals, academic researchers.

Dr. Heatwole will provide a detailed report on the results of this 6 month, randomized clinical trial of 42 participants providing data on safety and efficacy of mexiletine in reducing myotonia, improving muscle strength and lean muscle mass and other endpoints.

This session will provide results of several recent studies examining disease burden in myotonic dystrophy, including: the Christopher Project, a paper-based international patient-report survey presented by Dr. Katharine Hagerman; a burden of disease study funded by MDF and conducted by Mayo Clinic and Optum Labs, presented by Dr. Oliver Tobin, Mayo Clinic; patient insights provided through the 2016 MDF Patient-focused Drug Development Meeting and the 2017 MDF Annual Conference session "Bringing the Patient Voice to CNS-targeting Drug Development in DM", presented by Dr. Elizabeth Ackermann, Myotonic Dystrophy Foundation; and overview of the CDC's MD STARnet methodology, goals, and future directions as it pertains to myotonic dystrophy presented by Natalie Street.

Breath Easy: Pulmonary Symptom Management
Presenter Dr. Valeria Sansone, MD: Dr. Sansone will provide an overview of how DM can affect the respiratory system and how affected individuals can manage these symptoms.

MDF-Wyck Fellows present their current research in a Poster Session.

Industry and academic researchers and MDF-Wyck Foundation Fellows present posters on their current research projects.

Becoming a DM Advocate: Community Training
Instructor Kevin Brennan: Join MDF public policy consultant and advocacy expert Kevin Brennan to learn you can help raise the profile of myotonic dystrophy with members of Congress and advocate for important research and care legislation.

This panel presentation will review current efforts to use gene therapy and gene editing to treat myotonic dystrophy.

Adaptive Dance
Instructor Mindy Kim: Bring your dancing shoes and join community member Mindy Kim in a fun dance lesson specifically designed for people living with myotonic dystrophy.

Join community members and DM professionals at the opening reception, taking place in the beautiful Water's Edge Bar, located at water level in the Delta Pavillion among lush plants and waterfalls. Hors d'oeuvres will be served and a cash bar will be available.

Dinner meeting for site professionals participating in the current DMCRN study.

Check in and receive your free conference bag! (Includes bracelets, a pen, a program, and networking cards)

Visit the Research & Vendor Showcase in the Governor's Lobby to talk to researchers about current studies, and talk with industry professionals, academic researchers and MDF-Wyck Fellows about their current research via the concurrent Poster Session.

A program designed specifically for juvenile-onset adults living with DM, facilitated by MDF community members Barry Cohen, Ph.D and Diane Bade, RN, CAVS.

This interactive panel session will be similar in format to the Patient-Focused Drug Development meeting presented by MDF as a part of the 2016 MDF Annual Conference, and the CNS Endpoint Development discussion presented at the 2017 MDF Annual Conference. Panelists will discuss the disease symptom changes they have experienced living with myotonic dystrophy, followed by facilitated audience participation. This session is intended to amplify and deepen our understanding of disease changes and progression from the perspective of individuals and families living with DM.

Following the interactive panel discussion on myotonic dystrophy disease changes and progression over time, Dr. Cynthia Gagnon will present current strategies for managing disease progression and maintaining quality of life, including the latest in tools and resources.

MDF has recently completed Consensus-based Care Recommendations for Adults with DM1, DM2 and congenital DM. Now we need to make sure that every clinician treating a DM patient has them and is using them – and we need your help! MDF is asking our community members to help educate their doctors about the care recommendations, and we'll show you how. Come watch DM doctors and community members act out entertaining skits that will explain what to say to your doctors during your doctors visits. These skits will include a twist: affected community members will be the doctors and doctors are patients in these hilarious educational vignettes.

Dr. Elizabeth Ackermann will report on the results of the 3-year MDF and Wyck Foundation-funded drug development acceleration effort, MDF 3.0, and introduce MDF’s new initiative, MDF 4.0, including the $1M gene editing project, the Myotonic Dystrophy Clinical Research Network study and other aggressive therapy development programs.

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field. Moderated by Dr. Elizabeth Ackermann, MDF Chief Science Officer.

A panel of clinical and research experts will answer community member questions that have been submitted throughout the conference.

A seated, multi-course dinner will be followed by a live DJ and dancing to close out the conference.

Lounge and Coffee
Enjoy coffee and a sitting area with the MDF community.

First Timers' Tea
A welcome session for first-time attendees. Learn tips for having a successful conference experience and get a personal mentor to help you navigate the two-day event. MDF Board members, support group facilitators and experienced attendees will present, and we’ll have a session cheat sheet to help you plan your experience. Beverages will be served.

Adaptive Chair Yoga with Ellen Shapiro
Come stretch and relax in a yoga session specifically designed for people living with myotonic dystrophy.

Research & Vendor Showcase
Visit the Research & Vendor Showcase to talk to researchers about current studies, meet the famous Service Dogs, academic researchers, local organizations and more.

Conference Welcome
The opening session will include updates on our Care and Cure programming and some exciting Foundation announcements.

DM101: Understanding the Disease Basics with Lauren Elman, MD
A session for first-time attendees and those interested in a refresher on DM genetics, symptom burden and disease management strategies.

Grassroots Advocacy-MDF Needs You! with Jenn M. Dale, MA
Join MDF’s public policy consultant and advocacy expert to learn how you can educate your members of Congress about myotonic dystrophy and advocate for important research and care legislation.

DM Genetics & Family Planning with Darren Monckton, PhD and Tanya Bardakjian, MS
Experts in DM genetics will discuss family planning and myotonic dystrophy, including the specifics of the genetic mutation, and practical considerations for affected community members considering starting a family.

Research Poster Session (During Welcome Reception)
MDF research Fellows, industry professionals and academic researchers present their current research and answer questions via 1:1 discussions at their posters. Grab a beverage and circulate!

Research & Vendor Showcase
Visit the Research & Vendor Showcase to talk to researchers about current studies, meet the famous Service Dogs, academic researchers, local organizations and more.

Juvenile-onset Adult (JOA) Program with Barry Cohen, PhD and Diane Bade, RN, CAVS
A program designed specifically for juvenile-onset DM adults, facilitated by MDF community members.

Listening Room
Want to share your story? Need someone to talk to? Take a break and connect with professional listeners.

Exercise, Nutrition & Speech with Katy Eichinger, PhD, DPT, Leslie Vnenchak, MA, CCC-SLP, and Robin Meyers, RD, MPH, LDN
This rehabilitation panel will review current research and strategies for managing DM symptoms through exercise, speech and nutrition.

DM and Sleep: What's New, What Works with John Day, MD, PhD
Learn about current research and management strategies regarding myotonic dystrophy sleep issues, including disregulated sleep patterns and excessive daytime sleepiness.

Cardiac General Session with Saman Nazarian, MD, PhD
This session will explains how DM may impact your heart, and gives an overview of the cardiac electrical system, common symptoms associated with conduction problems, and preventative measures.

Patients Report Out: Living with DM over 50 with Ericka Simpson, MD
This interactive panel session will be similar in format to the Living with DM: Patient Report on Changes Over Time that was presented at the 2018 MDF Annual Conference. Panelists will discuss the disease symptom changes they have experienced living with myotonic dystrophy, followed by facilitated audience participation. This session will help researchers and drug developers understand disease changes and progression from the perspective of over-50 individuals living with DM and their families.

Industry Updates and Q&A with Industry Presenters TBD
Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field, including companies new to the MDF Annual Conference.

Closing Dinner & Dance Party
A seated, multi-course dinner will be followed by a live DJ and dancing to close out the conference.