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

Rebecca Ahrens-Nicklas

Personalized therapeutic genome editing for urea cycle disorders

0920-0940

Open discussion

0940-0955

Break

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

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

1115-1135

Johannes Häberle

Diagnostic and clinical trial applications of 

in vivo ureagenesis measurement

1135-1155

Open discussion

1155-1255

Lunch

Time

Speaker

Topic

Moderators: 

1255-1305

Tresa Warner 

Barbara Yu 

Innovative therapies: Patients' expectations 

1305-1325

Yoko Nakajima

Dietary management of urea cycle disorders: A comparative perspective from Japan and the United States

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