2024 PFIC Family & Scientific Conference

Join us in Cincinnati!

April 25-27, 2024

The PFIC Family & Scientific Conference is back! This unique conference brings together PFIC patients, families, clinicians, researchers, and industry stakeholders for an unforgettable experience of learning, sharing, and networking.

We are partnering with the Cincinnati Children’s Hospital and Medical Center to host this year’s conference. We are excited to work together to make this event a special one. We hope you can join us!


Presenting Sponsors

Gold Sponsor


Registration

There is still time left to register for the conference, both in person and virtually. Visit our registration page to find out more and register today!


Conference Agenda

The conference format includes family-oriented sessions, research workshops, and combined roundtable discussions. Family-oriented sessions will include informative talks by field experts with ample time for audience Q&A. These talks will be complemented by immersive roundtable discussions that foster collaborative dialogue between patients, families, clinicians, and researchers. The research workshop sessions are designed for researchers, physicians and industry to share and learn about new disease models, connect with each other to build next projects and to highlight problems important to patients.

Topics

Family Session Topics

  • Current & Emerging Treatments for PFIC
  • Current Prognosis and Prospects of Liver Transplant
  • Pre-Transplant Diet & Nutrition
  • Patient Centered Outcomes & the PFIC Network Patient Registry
  • PFIC in Adults
  • Future Treatments for PFIC

Research Workshop Topics

  • Novel PFIC2 models
  • Models for new types of PFIC
  • PFIC1 model and gene silencing therapies for Alagille Syndrome
  • Gene and cell therapies

Roundtable Discussion Topics

  • Project IMPACT and Patient Centered Outcomes Research
  • Understanding Risk/Benefit of PFIC Treatments, Present and Future

Conference Speakers

We’ve got an incredible roster of PFIC experts lined up for the conference this year.

Schedule

There will be a welcome dinner on Thursday, April 25th and an optional family activity to the Cincinnati Zoo on Friday, April 26th. The zoo activity will take place during the first research workshop session. Families will have the choice to attend either activity. Dinner will be hosted at the Cincinnati Zoo on Friday evening.

Arrival (Thursday April 25th, 2024)

Conference Welcome Reception: The Graduate Hotel, 7-9 PM

Day One (Friday April 26, 2024)

7:00 -8:00 amBreakfast and Registration
8:00 – 10:00amPlenary & Family Oriented Sessions:

Welcome Address/Opening Remarks
Current & Emerging Treatment Landscape (P. Hertel)
PFIC in Adults: Diagnosis & Onset (S. Vilarinho)
10:00-10:20amBreak
10:20-11:30amPre-Transplant Diet & Nutrition Panel Presentation & Discussion
(R. Sokol, E. Tamayo, Patient & Parent Representatives, Moderator: L. Bull)
Patient-Centered Outcomes & the PFIC Network Patient Registry
(G. Lubke)
11:30-12:15pmLunch
12:15-2:15pmFamily-Researcher Roundtable #1
“Patient-Centered Outcomes Research Priorities in PFIC”
-Facilitators: J. Squires, A. Perez (PFIC parent)
-IMPACT Project Team Members: G. Lubke, M. Kochanowsky, P. Catalla
2:15-2:30pmBreak
2:30 – 4:10 pmOptional Family Activity: free Cincinnati Zoo visit @ 3pm
Research Workshop:
Keynote Address: Understanding and mitigating hepatic bile acid accretion in pediatric cholestatic diseases (S.Karpen)
-A rapid mouse model system for PFIC by CRISPR-based multigenic knockout (H.Hayashi)
Novel mouse models for PFIC (H. Verkade)
4:10-4:20 pmBreak
4:20-5:15 pmResearch Workshop:
IPSC Model-New Type of PFIC (S. Vilarinho)
Using complementary experimental models to study new PFIC genes and variants (C.Yin)
6:30-8 pmDinner @ Cincinnati Zoo

Day Two (Saturday April 27th, 2024)

7:00 – 8:00 amBreakfast and Registration
8:00 – 9:00amResearch Workshop:

FIC1-ko iPSC model (R. Florentino)
Alagille model and development of new RNAi therapy (S. Huppert)
9:00-9:10amBreak
9:10-10:10amIn vivo selection for cell and gene therapy in the liver (M. Grompe)
Gene therapy targeting cholangiocytes (H. Willenbring)

10:10-10:30 amBreak
10:30-11:10amPlenary & Family Oriented Sessions:
State of the Art: Current Prognosis and Prospects of Liver Transplant
( H. Verkade)
11:10-11:30pmGroup Picture
11:30 – 12:30pm
Lunch
12:30-1:10pmFuture Treatments of PFIC (A.Asai)
1:10-1:20pmBreak
1:20-3:20pmFamily-Researcher Roundtable #2
“The Benefits and Risks of PFIC Clinical Research”
-Facilitators: R. Thompson, L. Susoi (PFIC parent)
3:20-3:35pmBreak
3:35-4:20pmPlenary & Family Oriented Sessions:
Summary Report of Research Workshop (C. Yin, W. Balistreri)
Closing Remarks
(E. Ventura)

*Times and running order subject to change

Continuing Education

In support of improving patient care, this activity has been planned and implemented by Cincinnati Children’s and PFIC Network. Cincinnati Children’s is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

  • CME: This activity has been approved for AMA PRA Category 1 Credit™.
  • Nursing: This activity is approved for continuing nursing education (CNE) contact hours.
  • IPCE: This activity was planned by and for the healthcare team, and learners will receive Interprofessional Continuing Education (IPCE) credits for learning and change.

If you need proof of attendance or credit, please visit the CCHMC CME portal to make your profile to receive credit.

Fun Things To Do In Cincinnati:

FC Cincinnati MLS Soccer Game– Saturday April 26th @ 7:30pm

  • Join us for an informal post-conference soccer game! The CCHMC team has reserved a block of seats for Saturday night’s FC Cincinnati game and you are invited to attend. Tickets are $46 per person. Space is limited, so if you are interested, please sign up here!

Cincinnati Zoo

  • The world famous Cincinnati Zoo is less than a mile from the conference venue and hotel.

Smale Riverfront Park

  • This park connects downtown Cincinnati to the Ohio River. It features multiple water play features, giant swings, an interactive foot piano, an imaginative play area for kids, expansive pollinator gardens, and Carol Ann’s Carousel. Several historical monuments can be found like the Black Brigade Monument and the recently dedicated Marian Spencer Statue.

Newport Aquarium

  • In the same area as the aquarium, there is also a LEGO cafe, playground, movie theater, and lots of restaurants.

Cincinnati Museum Center

  • Cincinnati Museum Center is a one-of-a-kind, multi-museum complex housed in Union Terminal, a historic Art Deco train station and National Historic Landmark. The complex includes the Cincinnati History Museum, the Cincinnati History Library and Archives, The Children’s Museum, the Museum of Natural History & Science and the Robert D. Lindner Family OMNIMAX® Theater.

Cincinnati Reds Great American Ball Park Tour

  • Get an inside look at the home of the Cincinnati Reds! Booking ahead of time is recommended

Findlay Market

  • Findlay Market is Ohio’s oldest continuously operated public market and one of Cincinnati’s iconic institutions. It is home to more than 50 full-time merchants selling meat, fish, poultry, produce, flowers, cheese, deli, ethnic foods and more.

Washington Park

  • A historic park in downtown Cincinnati with a great playground, interactive fountains and a patio with a bar.

Conference Lodging:

There is a conference room block reserved at The Graduate Cincinnati. Hotel rooms are available at a discounted conference rate when booked through the PFIC Network room block. It is located in downtown Cincinnati, just minutes from the conference venue. 

Scholarship Opportunities for PFIC Patients and Families:

The application period for Family Travel Scholarships is now closed. All applicants will be updated on the status of their application by April 12th and must register for the conference by April 15th.

If you are a US resident, the National Organization for Rare Disorders (NORD) offers several scholarships to help cover travel costs that rare disease patients and caregivers may apply for:

Event Health and Safety:

We are taking precautions to make members of our at-risk community feel safe attending in person if they so choose. This event will abide by all local and venue rules and regulations regarding masking, vaccination and disease prevention. We encourage all attendees to get vaccinated, take a Covid test before traveling and to attend virtually if feeling ill.