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2019 MDF Annual Conference Agenda

Friday, September 14

  • 7:30 am
    Breakfast (Professionals)
  • Programs
    Drug Development Roundtable - Natural History Data Updates (Professionals)
    Moderator: Kathie Bishop, PhD, Ami Mankodi, MD - Results from the Multicenter Observational Study of DM1, Nicholas Johnson, MD - Update on the Trial Readiness and Endpoint Assessment in cDM1 Study, Cynthia Gagnon, PhD - Results from a 9-Year Longitudinal Study in DM1 Patients
    Click for Details
    • 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.

    9:00 am
  • Registration Session
    Friday Registration
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    • Check in and receive your free conference bag! (Includes bracelets, a pen, a program, and networking cards)

    5:30 pm
  • Social Program
    Support Group Facilitators Breakfast (Closed)
    MDF Program Director
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    • A closed meeting for MDF Support Group Facilitators, hosted by the MDF Program Director. Brunch will be provided.

    9:50 am
  • Programs
    Population-Based Prevalence Study in DM1 & DM2 (Professionals)
    Moderator: Kathie Bishop, PhD, Nicholas Johnson, MD -Population-Based Prevalence Study in DM1 & DM2
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    • 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.

    9:20 am
  • Programs
    Current Advances in DM2 Research (Professionals)
    Moderator: Laura Ranum, PhD, Andy Berglund, PhD - Blocking Transcription in DM2, Chad Heatwole, MD - Measuring Patient-Reported Disease Burden in DM2, Laura Ranum, PhD - RAN Translation and DM2, Charles Thornton, MD - STOPP-DM2, Lukasz Sznajder, PhD - Intron Retention in DM2
    Click for Details
    • 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.

    11:00 am
  • Social Program
    First Timers' Tea
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    • 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.

    10:50 am
  • 11:00 am
    Coffee Break (Professionals)
  • Programs
    Molecular & Imaging Biomarker Development for DM Studies (Professionals)
    Moderator: Charles Thornton, MD, Donovan Lott, PhD - Development of Muscle MRI Endpoints as a Potential Biomarker for DM1, Peg Nopoulos, PhD - MRI Studies on Brain Structure and Function in DM1 Patients, Charles Thornton, MD - Updates on the Development of a Muscle Splicing Biomarker Assay, Thurman Wheeler, MD - Extracellular RNA as a Biomarker for Myotonic Dystrophy
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    • 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.

    12:30 pm
  • 12:30 pm
    Lunch (Professionals)
  • Programs
    Conference Welcome
    Molly White, CEO, MDF
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    • The opening session will include a welcome and Care and Cure progress update from MDF CEO Molly White.

    1:00 pm
  • Programs
    Breakout Sessions: Congenital Community Panel; The DM2 Patient Experience Community Panel; Gut Instincts: GI Symptom Management
    Leila Neshatian, MD, MSc, Stanford University
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    • Congenital Community Panel
      Facilitator TBD: Facilitated support group discussion for caregivers of congenital and childhood-onset myotonic dystrophy patients.

    • The DM2 Patient Experience: Community Panel
      Facilitator TBD: DM2 patients and caregivers will discuss symptom burden, unique aspects of the DM2 disease genotype and management strategies.

    • 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.

    1:50 pm
  • Programs
    JOA Program
    Barry Cohen, PhD, Diane Bade, RN, CAVS
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    • A program designed specifically for juvenile-onset adults, facilitated by MDF community members Barry Cohen, Ph.D and Diane Bade, RN, CAVS.

    4:50 pm
  • Poster Session
    Research & Vendor Showcase
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    • Visit the Research & Vendor Showcase in the Governor's Lobby to talk to researchers about current studies, and talk with industry professionals, academic researchers.

    3:50 pm
  • Programs
    Clinical Efficacy Trial of Mexiletine for DM1 (Professionals)
    Moderator: Thomas Cooper, MD, Chad Heatwole, MD - Clinical Efficacy Trial of Mexiletine for DM1
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    • 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.

    1:50 pm
  • Programs
    DM Disease Burden: Results from Recent Research (Professionals)
    Moderator: Thomas Cooper, MD, Oliver Tobin, MD (Mayo/Optum Project) - Myotonic Dystrophy Burden of Disease Analysis, Katharine Hagerman, PhD (Christopher Project) - Report from the Christopher Project, Elizabeth Ackermann, PhD - Myotonic Family Registry Update and Outcomes from MDF CNS Workshop, Natalie J. Street, MS, CGC - Updates on Myotonic Dystrophy Data Collection in the MD-STAR Net
    Click for Details
    • 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.

    3:00 pm
  • Programs
    Breakout Sessions: DM 101: Getting a Handle on the Basics; Breath Easy: Pulmonary Symptom Management; Male Caregivers Community Panel; Female Caregivers Community Panel
    Valeria Sansone, MD, University of Milan, NEMO Center, Nicholas Johnson, MD, University of Utah
    Click for Details
    • Male Caregivers Support Session
      Facilitator TBD: Closed session for male caregivers

    • Female Caregivers Support Session
      Facilitator TBD: Closed session for female caregivers

    • DM 101: Getting a Handle on the Basics
      Nicholas Johnson, MD, University of Utah. Session for first-time attendees and those interested in a refresher overview of DM genetics, symptom burden and management strategies.

    • 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.

    2:50 pm
  • Poster Session
    Poster Session (Professionals)
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    • Industry and academic researchers and MDF-Wyck Foundation Fellows present posters on their current research projects.

    4:00 pm
  • Poster Session
    Poster Session
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    • MDF-Wyck Fellows present their current research in a Poster Session.

    3:50 pm
  • Programs
    Breakout Sessions: Patient Report Out on Cannabis for Symptom Management; Staying Strong: DM and Exercise; Becoming a DM Advocate: Community Update and Training
    Katy Eichinger, PT, DPT, NCS, University of Rochester, Kevin Brennan, Faegre Baker Daniels
    Click for Details
    • Patient Report Out on Cannabis for Symptom Management
      Facilitator TBD: Many MDF community members and some DM clinical professionals report benefit from the use of medical cannabis to treat DM symptoms, including pain, anxiety, sleep issues and myotonia. In this moderated session, people living with DM will share their experiences using medical cannabis via audience feedback on a specific set of questions.

    • DM and Exercise
      Presenter Dr. Katy Eichinger, PhD: Dr. Eichinger will discuss current research and science on exercise to manage DM symptoms and improve quality of life now. An ongoing favorite at the MDF Annual Conference.

    • 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.

    4:50 pm
  • Programs
    Gene Editing and Therapy Development (Professionals)
    MODERATOR: Darren Monckton, PhD, Darren Monckton, PhD - Summary of the MDF Gene Editing Workshop, Eric Wang, PhD - Gene Therapy and Gene Editing for DM - Progress, Promise, and Considerations, Ranjan Batra, PhD - Transcriptome Editing for Therapeutic Treatment of Myotonic Dystrophy
    Click for Details
    • This panel presentation will review current efforts to use gene therapy and gene editing to treat myotonic dystrophy.

    5:00 pm
  • 5:00 pm
    Break
  • General Session
    Chair Tap Adaptive Dance Class
    Mindy Kim
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    • 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.

    5:50 pm
  • Social Program
    Opening Reception
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    • 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.

    8:00 pm
  • Programs
    DMCRN Dinner Meeting (Site PIs, PTs, CRCs only, Professionals)
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    • Dinner meeting for site professionals participating in the current DMCRN study.

    8:00 pm

Saturday, September 15

  • 7:00 am
    Breakfast
  • Registration Session
    Saturday Registration
    Click for Details
    • Check in and receive your free conference bag! (Includes bracelets, a pen, a program, and networking cards)

    5:00 pm
  • General Session
    Living with DM: Patients Report on Changes Over Time
    Moderator: Charles Thornton, MD, University of Rochester
    Click for Details
    • 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.

    10:50 am
  • Poster Session
    Research & Vendor Showcase 2
    Click for Details
    • 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.

    3:20 pm
  • General Session
    JOA Program 2
    Barry Cohen, Ph.D., Diane Bade, RN, CAVS
    Click for Details
    • 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.

    2:20 pm
  • General Session
    Strategies for Managing Progression
    Cynthia Gagnon, OT, PhD, Missy Dixon, PhD
    Click for Details
    • 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.

    11:50 am
  • 12:00 pm
    Lunch
  • General Session
    DM2 Research Update Lunch
    Chad Heatwole, MD
    Click for Details
    12:50 pm
  • General Session
    Consensus-based Care Recommendations: What Do They Mean for You?
    Nicholas Johnson, MD, University of Utah, Jacinda Sampson, MD, PhD, Stanford University, Missy Dixon, PhD, University of Utah
    Click for Details
    • 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.

    2:20 pm
  • 2:20 pm
    Break
  • General Session
    Accelerating the Search for Therapies: What's Happening, What's Next
    Elizabeth Ackermann, PhD
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    • 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.

    3:15 pm
  • General Session
    Conference Challenge
    Click for Details
    3:30 pm
  • General Session
    Industry Updates on Drug Development
    Laurence Mignon, PhD, Ionis Pharmaceuticals, Matthew Disney, PhD, Expansion Therapeutics, Ranjan Batra, PhD, Locana Bio, Joseph P. Horrigan, MD, AMO Pharma Ltd.
    Click for Details
    • 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.

    4:50 pm
  • General Session
    Conference Q&A
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    • A panel of clinical and research experts will answer community member questions that have been submitted throughout the conference.

    5:15 pm
  • 5:15 pm
    Break - No Host Cocktail Hour
  • Social Program
    Closing Dinner, Line Dancing Class & Dance Party
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    • A seated, multi-course dinner will be followed by a live DJ and dancing to close out the conference.

    8:00 pm

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