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2019 MDF Annual Conference Agenda

Friday, September 13

  • Closed Professional Session
    DM Biomarkers: Goals, What's Working & How They'll Be Used for Drug Development & Approval
    Moderator: Charles Thornton, MD, University of Rochester
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    • DM Biomarkers: Goals, What's Working & How They'll Be Used for Drug Development & Approval with:
      • Frank Bennett, PhD, Ionis Pharmaceuticals
      • Katy Eichinger, PhD, DPT, University of Rochester
      • Donovan Lott, PhD, University of Florida
      • Peg Nopoulos, PhD, University of Iowa
      • Matthew Tanner, MS, University of Rochester
      • Thurman Wheeler, MD, Massachusetts General Hospital
      8:30am - 9:45am
    9:45 am
  • 9:45 am
    Break
  • Closed Professional Session
    Current Therapeutic Approaches
    Moderator: Doug Kerr, MD, PhD, MBA, Generation Bio
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    • Current Therapeutic Approaches with:

      • Andy Berglund, PhD, RNA Institute
      • Vincent Dion, PhD, University of Wales
      • Matthew Disney, PhD, Scripps Research Institute/Expansion Therapeutics
      • Steve Finkbeiner, MD, PhD, Gladstone Institutes
      • Paolo Fortouna, MD, PhD, Roche, Invited
      • Darren Monckton, PhD, University of Glasgow
      • Christopher Pearson, PhD, Hospital for Sick Children
      • Catherine Stehman-Breen, MD, Dyne Therapeutics
      • Gene Yeo, PhD, UCSD/Locana Bio
      • Other speakers TBD
      10:00am - 12:00pm
    12:00 pm
  • 12:00 pm
    Lunch (Provided)
  • Closed Professional Session
    FDA: Current Thinking on the Orphan Disease Regulatory Pathway; Insights on Surrogate Biomarkers, CNS and Muscle Endpoints, Accelerated Approval
    Billy Dunn, MD, Center for Drug Evaluation and Research, (Invited)
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    • FDA: Current Thinking on the Orphan Disease Regulatory Pathway; Insights on Surrogate Biomarkers, CNS and Muscle Endpoints, Accelerated Approval with Billy Dunn, MD, Center for Drug Evaluation and Research, Invited

      12:30pm - 1:00pm
    1:00 pm
  • Closed Professional Session
    DM2 State of the Science: Current Research, Next Steps
    Moderator: Laura Ranum, PhD, University of Florida
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    • DM2 State of the Science: Current Research, Next Steps with Allison Formal, MBA

      1:00pm - 1:30pm
    1:30 pm
  • Closed Professional Session
    Study and Trial Learnings to Date – Observations on Design, Endpoints & Patient Enrichment Strategies
    Moderator: Kathie Bishop, PhD, Otonomy
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    • Study and Trial Learnings to Date – Observations on Design, Endpoints & Patient Enrichment Strategies with:

      • Guillaume Bassez, MD, PhD, Institut de Myologie
      • Chad Heatwole, MD, University of Rochester
      • Joseph Horrigan, MD, AMO Pharma Ltd.
      • Laurence Mignon, PhD, Ionis Pharmaceuticals
      • Christopher Turner, MD, University College London
      1:30pm - 3:00pm
    3:00 pm
  • 3:00 pm
    Break
  • Closed Professional Session
    PRMRP: Specifics of This International Research Funding Program and Strategies for Application
    Moderator: TBD
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    • PRMRP: Specifics of This International Research Funding Program and Strategies for Application with Patricia Modrow, PhD, US Department of Defense, Invited

      3:15pm - 3:45pm
    3:45 pm
  • Closed Professional Session
    DM Animal Models: What We Have, What We Need and Why
    Moderator: Thomas Cooper, MD, Baylor College of Medicine
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    • DM Animal Models: What We Have, What We Need and Why with:

      • Laurent Bogdanik, PhD, The Jackson Labs
      • Sharon Hesterlee, PhD, Lion Therapeutics
      • Laura Ranum, PhD, University of Florida
      • Ashish Rao, PhD, Baylor College of Medicine
      • Charles Thornton, MD, University of Rochester
      • Eric Wang, PhD, University of Florida
      • Thurman Wheeler, MD; Massachusetts General Hospital
      3:45pm - 5:00pm
    5:00 pm
  • 5:00 pm
    Break
  • Social Program
    Welcome Reception & Research Poster Session
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    • Welcome Reception
      Join community members and DM professionals at the opening reception. Hors d'oeuvres will be served and a cash bar will be available. Be sure to visit the research poster session that will occur during the reception.

      5:30pm - 7:30pm
    • Research Poster Sessions (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!

      5:30pm - 7:30pm
    7:30 pm

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