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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 the care and cure of patients wtih myotonic dystrophy. 

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

Agenda


Time

Presentation

8:30 AM

Welcome: 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

Break


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

Lunch


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

Adjourn

 

 

 

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