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

Location: Carey Auditorium at Hesburgh Library

Rare Disease Invite 10-12-2018


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 Welcome: John 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 Stamler, CEO of the Harrington Drug Discovery Institute

                                    Steve Aselage, CEO, Retrophin

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

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

Kasturi 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 Gershkowitz, Chief Patient Advocate, Amicus Therapeutics

Panelists | Nicole Boice, Founder, Global Genes

                           Cindy Parseghian, Ara Parseghian Medical Research Fund

Jean Campbell, Principal, 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)



The Rare Disease Symposium will conclude after lunch on Friday. However, attendees are welcome to participate in the following Ara Parseghian Medical Research Fund (APMRF) activities.

Friday, October 12  | Parseghian Cup Rugby Match (Open to the Public) | Stinson Rugby Stadium

5:00–6:30 PM | For the past six years, the University of Notre Dame Men’s Rugby team has played the University of Arizona in an annual match called the Parseghian Cup. The universities take turns hosting the event and this year, the Parseghian Cup will be played at Notre Dame The Parseghian Cup is open to the entire campus, and we hope to have an enthusiastic crowd watch the Irish and Wildcats go head-to-head.

Saturday, October 13

2:30 PM | Notre Dame vs Pittsburgh Football Game | Tickets may be available at 



Sponsor Logos (rare disease event)




Accommodations (attendees must make their own reservations)

Please note: hotels fill quickly and rates vary widely during football season. The list of hotels provided below does not guarantee that rooms are available and no room blocks have been reserved at any hotel for this event. Please call each hotel for availability and rates. 

Doubletree 574-234-2000

Hampton Inn & Suites (South Bend) 574-277-9373

Courtyard by Marriott 574-237-7777

Aloft 574-288-8000

Hampton Inn & Suites (Granger) 574-243-4600

Embassy Suites 574-400-2600

Fairfield Inn 574-234-5510

Hilton Garden Inn 574-232-7700