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

    5:00 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


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