TRNDS 2019 Agenda
Friday, September 13, 2019 | University of Rochester
7:30 – 8:15 am
Registration & Networking Breakfast
8:15 – 8:30 am
Welcome
Advancing Therapeutics for Rare Neurological Diseases
Erika Augustine MD, MS
Co-Chair, TRNDS; Associate Director, Center for Health + Technology (CHeT), University of Rochester
Scott Steele PhD
Co-Chair, TRNDS; Director, Regulatory Science Programs, Clinical & Translational Science Institute (CTSI), University of Rochester
John J. Foxe PhD
Kilian J. & Caroline F. Schmitt Chair in Neuroscience; Director, The Ernest J. Del Monte Institute for Neuroscience; Chair, Department of Neuroscience; Director, Center for Advanced Brain Imaging & Neurophysiology, University of Rochester
8:30 – 9:00 am
Keynote Address
Rare Disease as an Emerging Field of Medicine
Marshall Summar MD
Director, Rare Disease Institute, Children’s National Health System and Board Chair, National Organization for Rare Disorders
9:00 – 10:00 am
Panel Discussion
Innovation & Therapeutic Discovery
Sharon Begley BA
Senior Science Writer, STAT News (moderator)
Wilson Bryan MD
Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation & Research, U.S. Food & Drug Administration
David Jacoby MD, PhD
Vice President, Head of Clinical Science, BioMarin Pharmaceutical
Rachel McMinn PhD
President & CEO, Neurogene Inc.
Traci Schilling MD
Executive Director, Gene Therapy Clinical Development, PTC Therapeutics
10:00 – 10:30 am
Networking Break
10:30 – 10:45 am
Research Insights
Novel Therapies & Ethical Considerations
Holly Tabor PhD Associate Professor, Stanford University
10:45 – 11:45 am
Case Studies in Personalized Medicine
Martin Graham PhD
CEO, Empire Discovery Institute (moderator)
Tanya Fischer MD, PhD
Global Project Head, Sanofi-Genzyme
Jinkuk Kim PhD
Research Fellow, Division of Genetics & Genomics, Harvard Medical School
11:45 am – 1:00 pm
Lunch with Parallel Breakout Sessions
Breakout #1
Engagement & Patient-Centered Trials
Tauna Batiste MS
Executive Director, Batten Disease Support & Research Association
Danielle Edwards BA
Community Engagement Manager, Jett Foundation
Breakout #2
Paying for Orphan Drugs
Mona Chitre PharmD
Chief Pharmacy Officer & Vice President, Integrated Clinical Strategy & Innovation
Sean Nicholson PhD
Professor, Department of Policy Analysis & Management, Cornell University
1:00 – 2:00 pm
Panel Discussion
Access to Novel Therapies
Elissa Orlando MPA
Strategic Liaison, Clinical & Translational Science Institute, University of Rochester Medical Center (moderator)
Alison Bateman-House MPH, PhD
Assistant Professor, Department of Population Health, New York University
PJ Brooks PhD
Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health
Emily Farkas PA-C
Patient Advocate
Jayne Gershkowitz BA
Chief Patient Advocate, Amicus Therapeutics
2:30 – 2:45 pm
Research Insights
Measuring Cognitive Impairment in the Real World
Nikki Marinsek PhD
Data Scientist, Evidation Health
2:45 – 3:45 pm
Panel Discussion
Real World Evidence
Karl Kieburtz MD, MPH
Professor of Neurology, University of Rochester Medical Center (moderator)
Derek Bowen BS
Vice President Business Development & Strategy, Blackfynn
Ben Schlatka MBA
CEO and Co-Founder, MC10
Nancy Yu BS
Co-Founder & CEO, RDMD
Dina Zand MD
Medical Officer, Center for Drug Evaluation & Research, U.S. Food & Drug Administration
3:45 – 4:00 pm
Closing Remarks