Patient-Centered Therapy Development Meeting

Myotonic Dystrophy Patient Centered Therapy Development Meeting

September 17, 2015
Ritz-Carlton Pentagon City, Arlington, VA

Goal: Explore myotonic dystrophy (DM) heterogeneity and the development of meaningful and measureable DM clinical endpoints, dientifying biomakrers nad patient-reported outcmes to guide and advance the discovery of new therapies and design of clinical trials for patients living with myotonic dystrophy. 

Note: as additional presentations become available, MDF will upload them and link them below.




8:30 AM

Molly White - Chief Executive Officer, MDF

8:35 AM

Introduction of Chair
Woodie Kessel, MD, MPH, MDF Vice Chair

8:40 AM

Meeting Overview
Stephen Spielberg, MD, PhD, Editor-in-Chief of Therapeutic Innovation & Regulatory Science Journal

8:45 AM

FDA Perspective
Richard Moscicki, MD, Deputy Center Director for Science Operations, CDER, FDA

9:00 AM

Patient Perspectives and Disease Overview
Sarah Clarke, MD
Living with DM Short Film

9:15 AM

Disease Symptoms & Pathology Overview
Nicholas Johnson, MD, University of Utah

9:30 AM

Session 1:

10:30 AM

Discussion Panel: Session 1

11:00 AM


11:15 AM

Session 2: DM Trial Endpoint Selection

  • University of Rochester Natural History Study Data
    Charles Thornton, MD, University of Rochester
  • University of Sherbrooke Natural History Data, OMMYD Process & SOPs
    Cynthia Gagnon, PhD, University of Sherbrooke
  • Myotonic Dystrophy Health Index (MDHI)
    Chad Heatwole, MD, University of Rochester
  • Endpoint Selection & Validation
    Nikunj Patel, PhD, FDA

12:15 PM

Discussion Panel: Session 2

12:45 PM


1:30 PM

Session 3: FDA Case Studies

2:00 PM

Discussion Panel: Session 3

2:30 PM

Session 4: Candidate DM Biomarkers

  • The Big Picture: DM Biomarker Development
    John Day, MD, PhD, Stanford University
  • Current Thinking: DM Biomarkers for Clinical Trials
    John Carulli, PhD, Biogen Idec
  • Biomarkers in Drug Development
    Shashi Amur, PhD, FDA

3:00 PM

Discussion Panel: Session 4

3:30 PM

Summary and Next Steps Discussion
Steven Spielberg, MD, PhD

4:00 PM


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