Time 

Speaker 

Topic 

Moderators: Matthias Zielonka,  Andrea Gropman

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

Rebecca Ahrens-Nicklas

Personalized therapeutic genome editing for UCD

0920-0935

Open discussion

0935-0950

Break

Time

Speaker

Topic

Moderators: Kimihiko Oishi, Stefan Kölker

0950-1010

Andrea Gropman

Brain imaging as a prognostic biomarker for UCD

1010-1030

Greta Wilkening

Unraveling the neuropsychological and quality of life complexity of UCD

1030-1050

Matthias Zielonka

Early prediction of disease severity: 

a tool for precision medicine in UCD

1050-1110

Aimee Dudley

Large-scale functional assays for understanding variant impact in UCD

1110-1130

Johannes Häberle

Diagnostic and clinical trial applications of 

in vivo ureagenesis measurement

1130-1145

Open discussion

1145-1240

Lunch

Time

Speaker

Topic

Moderators: Carlo Dionisi-Vici, Nicholas Ah Mew

1240-1300

Tresa Warner 

Barbara Yu 

Innovative therapies: Patients' expectations 

1300-1320

Yoko Nakajima

An East-West comparison of UCD dietary management

1320-1340

Akinari Fukuda

Metabolic management versus whole liver 

or stem cell transplantation 

1340-1400

Cary O. Harding

Gene therapies for UCD

1400-1420

Gerald Schwank 

Current progress and challenges in gene editing for UCD

1420-1440

Sonam Gurung

RNA-based therapies for UCD: An update

1440-1455

Open discussion

1455-1510

Break

Time

Speaker

Topic

Moderators: Shirou Matsumoto, Giancarlo la Marca

1510-1530

Shawn McCandless

Promise and challenges of newborn screening for UCDs 

1530-1550

Yoichi Wada

Proteomic approach to screening for proximal UCDs

1550-1610

Stefan Kölker

Newborn screening for all UCD? Results of a pilot study

1610-1530

Ines Thiele

Opportunities in 

machine learning-supported newborn screening

1630-1645

Open discussion and wrap up