TRNDS 2019 Agenda

Friday, September 13, 2019 | University of Rochester

7:30 – 8:15 am

Registration & Networking Breakfast

8:15 – 8:30 am


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