July 16/17
Conference Main Lounge
23:00-23:15 Welcome & Introduction to PFIC
Network Presented by: Walter Perez, Tara Kearns, Emily Ventura
23:15-00:15 Keynote Speech #1: Basics of
Genetics, Introduction to PFIC as a set of Genetic
Disease of Cholestasis Presented by: Dr. William Balistreri, MD, Dr. Chunyue Yin, PhD
Saturday, July 17
00:30-01:30 What’s in the Research
- Introduction to research in PFIC, what barriers exist
Presented by Dr. Henkjan Verkade, MD
- NAPPED Study
Presented by Dr. Henkjan Verkade, MD
- What is known: Pruritus
Presented by Dr. Binita Kamath, MD
- The PFIC Patient Registry in partnership with CoRDs
01:40-02:40 Scientific Discussions: Drug Development related to PFIC
Update on IBAT Inhibitors: BylvayTM (odevixibat) and Ensuring Patient Access to PFIC Therapy
Presented by: Christine Clemson, M.S.C.I., PhD. & Pamela Stephenson, Albireo Pharma
Update on ASBT Inhibitors: Maralixibat
Presented by: Pamela Vig, PhD, Mirum Pharmaceuticals
Discussions about Gene Therapy
Presented by: Pamela Vig, PhD, Mirum Pharmaceuticals
03:00-04:00 What is PFIC? For young patients (school age
& teens) Presented by: Dr. James Squires, MD
04:00-04:30 Peer Support & Engagement: Kids take over
the lounge
– Show & Tell / Arts & Crafts / Meet & Greet!
05:00-06:00 Pruritus & Nutrition in PFIC: “Back to the Basics”
Presented by: Dr. Ben Schneider
08:00-all morning *Lounge will stay open to replay recordings
of earlier sessions
July 17/18
Conference Main Lounge
23:00-00:00 Considerations related to COVID &
PFIC Presented by: Dr. Ron Sokol
Sunday, July 18
00:10-01:20 Ambassador Training, North America,
South America, Eastern Europe
- Emily Ventura, PFIC Network
- Swapna Khakani, Everylife Foundation
- Francesca Lombardozzi, PFIC Italia Network
- Bernadetta Skora, PFIC Network Polska
- Dania Nair & Andrew Scott, Global Liver Institute
- Michelle Wilkins, Children’s Liver Disease Foundation
- Sindee Weinbaum, European Liver Patient Association
01:30-02:30 Keynote Speaker #2: Genetics of PFIC
by Subtype Presented by: Dr. Richard Thompson, Dr. Laura Bull
03:00-03:45 Mental Health and Daily Life Concerns
Presented by: Nicola Sereni, Psicologist, Tara Kearns, LMSW, Emily Ventura, RN
04:15-05:15 Q&A Present Panel Discussion
Panelists: Dr. Ben Schneider, Dr. Richard Thompson, Dr. Laura Bull, Dr.
Henkjan Verkade, Dr. James Squires, Dr. Alexander Miethke, Dr. Chunyue
Yin, Dr. William Balistreri
05:30-06:30 Peer Support and Patient
Engagement Presented by: Parent/patient advisory board
06:30-07:00 Peer Support and Engagement
Patients and Caregivers Networking in Lounge
07:00-08:00 Inspiration, Motivation, Closing Remarks
08:00-all morning *Lounge will stay open to replay recordings
of earlier sessions
July 16/17
Global Ambassador Lounge
21:00-23:00
UK
Saturday, July 17
1:30-3:30
Netherlands
4:00-6:00
Germany
6:00-8:00
Canada
20:00-22:00
India
22:00-00:00
Pakistan
Sunday, July 18
2:30-4:30
Italy
4:30-6:30
Poland
8:30-10:30
Australia