Models for Accelerating Academic Rare Disease Research and Innovation: A Working Session Hosted by Notre Dame

Author: College of Science

20181011 200945

An interactive working session hosted by Notre Dame to explore how universities can accelerate rare disease research and discovery through innovation, entrepreneurship and cross-sector partnerships.

John Crowley, Chairman and CEO of Amicus Therapeutics and Founding Board Member of Global Genes
Marshall Summar, MD, Chief of the Division of Genetics and Metabolism and Director of Clinical Research at Children's National Medical Center, Chairman of the Board of the National Organization for Rare Disorders (NORD).

Friday, October 12, 2018 | Carey Auditorium at Hesburgh Library

These events are free and open to the public, but please RSVP

8:00–9:00 AM | Continental breakfast

9:00–9:15 AM | Co-Chairs WelcomeJohn Crowley and Marshall Summar, MD

9:15–9:45 AM | Keynote: The Promise of Innovative Gene Therapies for Rare Disease–Universities at the Hub of Ground-breaking Technologies | James Wilson, MD, PhD, Director, Gene Therapy Program; Rose H. Weiss Professor and Director, Orphan Disease Center, University of Pennsylvania

9:45–11:00 AM | Stakeholder Panel: Overcoming Barriers to Rare Disease Drug Development (titles and speakers subject to change): TBA

Moderators | John Crowley and Marshall Summar, MD

Panelists | Dr. Jonathan StamlerCEO of the Harrington Drug Discovery Institute

                                    Steve AselageCEO, Retrophin

                                    Nina DiPrimio, PhD, Director of R&D, Perlara

Ellen Wagner, Parent Project Muscular Dystrophy’s Board of Directors

Katsuri Haldar, PhD, Director, Boler-Parseghian Center for Rare and Neglected Diseases, University of Notre Dame

Brian Blagg, PhD, Director, Warren Family Center for Drug Discovery & Development, University of Notre Dame

11:00–11:30 AM | BREAK

11:30 AM–12:40 PM | Stakeholder Panel | New Models in Patient Advocacy: Rare Disease Community Partners in Drug Development

Moderators | Sean Kassen, PhD, Director of Ara Parseghian Medical Research Fund, Notre Dame and Jayne GershkowitzChief Patient Advocate, Amicus Therapeutics

Panelists | Nicole BoiceFounder, Global Genes

                           Cindy ParseghianAra Parseghian Medical Research Fund

Jean CampbellPrincipal, JF Campbell Consultants and co-founder, Professional Patient Advocates in Life Sciences (PPALS)

Jennifer Bernstein, JD, Executive VP, Horizon Government Affairs

1:00–2:00 PM | Closing Lunch | Jordan Hall of Science Reading Room (southeast corner of first floor)