PFIC Family & Scientific Conference 2023 Agenda
SNEAK PEAK!
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
Times to be announced
Friday, May 5
Keynote Address: Patient Centered Outcomes Research
Dr. Jim Squires, Children’s Hospital of Pittsburgh
The PFIC Network Patient Registry + 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
Liver Transplant in PFIC
Dr. Greg Tiao, Cincinnati Children’s Medical Center
Therapeutic Development 101: What it takes to develop a new therapy
Part 1: From concept through clinical trials (15 minutes) Presented by: Mirum Pharmaceuticals
Part 2: Submitting for regulatory approval and commercializing (15 minutes) Presented by: Albireo Pharmaceuticals
Taking Research from Bench-to-Bedside: Chaperones and PFIC + Q&A (virtual)
Dr. Thomas Falguières & Dr. Emmanuel Gonzales, Universitè Paris- Saclay
Mental Well-Being with a Rare Disease + Q&A
Caitlin Shneider M.A., Georgia State University
Updates from ChiLDReN + Q&A
Dr. Paula Hertel, Texas Children’s Hospital
Updates from the NAPPED Consortium
Antonia Felzen, MD/PhD candidate, University of Groningen, Netherlands
What’s with the Number? PFIC Subtypes and the Meaning of a Genetic Diagnosis
Dr. Richard Thompson, Kings College London
Full Panel Roundtable
All presenters, Open Q&A with discussion
Networking Reception