Materials

Agenda

Participant List

Presentations

Welcome and Housekeeping
Logan Donaldson, Forum for Collaborative Research

Introductory Remarks
Veronica Miller, Forum for Collaborative Research
Pam Vig, Mirum Pharmaceuticals
Henkjan Verkade, University of Groningen

Session I: Scientific, Community, and Developmental Updates

Biliary Atresia Update
Ekkehard Sturm, University of Tübingen
Christoph Slavetinsky, University of Tübingen
Claus Petersen, Hannover Medical School
Jennifer Lau, BARE

PFIC Update
Henkjan Verkade, University of Groningen
Emmanuel Gonzales, Paris-Saclay University
Emily Ventura, PFIC Network

Alagille Syndrome Update
Binita Kamath, SickKids
Karsten Baumgaertel, ALGS Alliance

Pediatric Transition to Adulthood Panel
Deirdre Kelly, University of Birmingham
Jorge Amil Dias, Hospital Lusíadas
Ruth De Bruyne, Ghent Univerist
Nitika Gupta, Emory University

Session II: Regulatory and Research Considerations

Regulatory session
Johannes Taminiau, EMA and moderated discussion

Natural History Usage, Post Approval Expectations, Open Workshop Planning
Open Moderated Discussion

Closing Remarks
Pam Vig, Mirum Pharmaceuticals
Henkjan Verkade, University of Groningen
Veronica Miller, Forum for Collaborative Research

* Indicates Remote Participation