Agenda: Sept 2, 2025, 8AM – 5 PM
Session 1: Disease models and mechanisms
Time | Speaker | Topic |
Moderators: | ||
0800-0820 | Andrea Gropman | The journey to the current UCD landscape |
0820-0840 | Ljubica Caldovic | Cellular models for UCD research |
0840-0900 | Nicola Brunetti-Pierri | Using animal models to understand UCD |
0900-0920 | Ayelet Erez | The urea cycle beyond ureagenesis(Not Yet Confirmed) |
0920-0940 | Open discussion | |
0940-0955 | Break |
Session 2: From phenotypic diversity to precision medicine
Time | Speaker | Topic |
Moderators: | ||
0955-1015 | Andrea Gropman | Brain imaging as a prognostic biomarker for UCD |
1015-1035 | Greta Wilkening | Unraveling the neuropsychological complexity of UCD(Not Yet Confirmed) |
1035-1055 | Matthias Zielonka | Early prediction of disease severity:a tool for precision medicine in UCD |
1055-1115 | Aimee Dudley | A multiplexed approach to understandingvariant impact in UCD(Not Yet Confirmed) |
1115-1135 | Johannes Häberle | Diagnostic and clinical trial applications ofin vivo ureagenesis measurement |
1135-1155 | Open discussion | |
1155-1255 | Lunch |
Session 3: Innovative vs established therapies: Advantages and limitations
Time | Speaker | Topic |
Moderators: | ||
1255-1305 | Tresa WarnerBarbara Yu | Innovative therapies: Patients‘ expectations |
1305-1325 | Yoko Nakajima | An East-West comparison of UCD dietary management |
1325-1345 | Akinari Fukuda | Metabolic management versus whole liveror stem cell transplantation |
1345-1405 | Cary O. Harding | Gene therapies for UCD: from historical perspectivesto future prospects |
1405-1425 | Gerald Schwank | Current progress and challenges in gene editing for UCD |
1425-1445 | Sonam Gurung | RNA-based therapies for UCD: An update |
1445-1455 | Tresa WarnerBarbara Yu | Innovative therapies: outcomes |
1455-1515 | Open discussion | |
1515-1530 | Break |
Session 4: Challenges of newborn screening for UCDs
Time | Speaker | Topic |
Moderators: | ||
1530-1550 | Shawn McCandless | Newborn screening for UCD: Benefits and limitations |
1550-1610 | Ines Thiele | Utilizing neglected data: Opportunities inmachine learning-supported newborn screening |
1610-1630 | Yoicho Wada | Proteomic approach to screening for proximal UCDs |
1630-1700 | Open discussion and wrap up |