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Agenda: Sept 2, 2025, 8AM – 5 PM

 

Session 1: Disease models and mechanisms 

 

Time 

Speaker 

Topic 

Moderators: 
0800-0820
Andrea Gropman
Tresa Warner
 
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 understanding 
variant impact in UCD

(Not Yet Confirmed)

1115-1135
Johannes Häberle
Diagnostic and clinical trial applications of 
in 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 Warner 
Barbara 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 liver 
or stem cell transplantation 
1345-1405
Cary O. Harding
Gene therapies for UCD: from historical perspectives 
to 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 Warner
Barbara 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 in 
machine learning-supported newborn screening
1610-1630
Yoicho Wada
Proteomic approach to screening for proximal UCDs
1630-1700
Open discussion and wrap up