PFIC Family & Scientific Conference 2023 Agenda
May 4-6 at Disney Coronado Springs in Orlando, FL and virtual
*The conference agenda is subject to change based on speaker availability, decided topics of conversation, or other topics that may take priority between now and the conference.*
Time
3:00 PM
5:00 PM
7:00 – 9:00 PM
Thursday, May 4
Patient/Family Volunteer Rally
Check In Time & Poster Session
Welcome Dinner & Icebreaker Activity – Room Lantana
Time
8:00 – 8:15 AM
8:15 – 8:45 AM
8:45 – 9:15 AM
9:15 – 10:00 AM
10:00 – 10:45 AM
Friday, May 5 – Room: Monterrey 1
Welcome Address
Keynote Address: Patient Centered Outcomes Research
Dr. Jim Squires, Children’s Hospital of Pittsburgh
Paired with: Alex Perez, Parent, Canada
PFIC Network Science & Research Program: Updates on the Registry, Grant Proposals, and the Science Blog + Q&A
Dr. Gitta Lubke, PFIC Network
Clinical Care for PFIC: What does “disease progression” mean, and how do you measure it? + Q&A
Dr. Benjamin Shneider, Texas Children’s Hospital
Paired with: Rachael Turek, Parent, USA
Liver Transplant in PFIC
Dr. Greg Tiao, Cincinnati Children’s Medical Center
Paired with: Brittany Brinson, Parent, USA
Short Break
10:45 – 11:15
30 Minutes
11:15 – 12:00 AM
12:00 – 12:45 PM
Therapeutic Development 101: What it takes to develop a new therapy
Part 1: From concept through clinical trials (15 minutes) Presented by: Lara Longpre, Mirum Pharmaceuticals
Part 2: Submitting for regulatory approval and commercializing (15 minutes) Presented by: Lauren Wilkinson, Albireo Pharmaceuticals
Paired with: Cadence Crompton, Patient, Canada
Taking Research from Bench-to-Bedside: Chaperones and PFIC + Q&A (virtual)
Dr. Thomas Falguières & Dr. Emmanuel Gonzales, Universitè Paris- Saclay
Paired with: Laura Susoi, Parent, Canada
Lunch Break
12:45 to 1:30
1:30 – 2:15 PM
2:15 – 3:00 PM
3:00 – 3:45 PM
3:45 – 4:30 PM
The psychosocial impact of living with a rare chronic disease on youth and their families + Q&A
Caitlin Shneider M.A., Doctoral Candidate, Georgia State University
Paired with: Cedar Ventura, Patient, USA
Updates from ChiLDReN + Q&A
Dr. Paula Hertel, Texas Children’s Hospital
Paired with: Rocio Cavazos, Parent, Mexico
Updates from the NAPPED Consortium
Antonia Felzen, MD/PhD candidate, University of Groningen, Netherlands
Paired with: Tara Kearns, Parent, USA
What’s with the Number? PFIC Subtypes and the Meaning of a Genetic Diagnosis
Dr. Richard Thompson, Kings College London
Paired with: Brittany Brinson, Parent, USA
Short Break
4:30 – 4:45
15 Minutes
4:45 – 5:30 PM
5:30 – 5:45 PM
6:00 – 8:00 PM
Full Panel Roundtable
All presenters, Open Q&A with discussion
Conference Debrief Activity (Closed session for Patients and Families)
Lisa Jensen to Lead
Networking Reception at Villa Del Lago
Time
8:00 – 9:00 AM
9:20 – 10:20 AM
Saturday, May 6
Science Engagement Focus Group (CLOSED)
PFIC Network Patient Registry Focus Group (CLOSED)