6th International Symposium on Urea Cycle Disorders

On September 1-2, 2025, in Kyoto, Japan, research on urea cycle disorders took center stage in front of the international research community as NUCDF co-chaired the 6th International Symposium on Urea Cycle Disorders, “Advancements in Understanding: Global Perspectives and Innovations in Urea Cycle Disorders.” The event was held as a satellite meeting to the International Congress of Inborn Errors of Metabolism (ICIEM). NUCDF's research partner, the Urea Cycle Disorders Consortium (UCDC), also co-chaired the meeting. 

NUCDF executive director Tresa Warner kicked off the event by delivering an an opening talk with Andrea Gropman, MD of St. Jude Children's Research Hospital, who is principal investigator of the UCDC. Warner also delivered a podium presentation on NUCDF activities and the patient experience, speaking alongside Barbara Yu, president of the Citrin Foundation. She will also be helping lead a separate session during the larger ICIEM meeting on quality of life in patients with inborn errors of metabolism, presenting with leaders from Metabolic Support UK and the MSUD Family Support Group. 

NUCDF also presented posters on several of the foundation's recent initiatives:

• Urea Cycle Disorders ECHO, The Essentials: Educating front-line care providers, expanding the expert network
• Validating MyRareDiet®: A diet tracking and mHealth tool for patients with inborn errors of metabolism
• The NUCDF Partner Network: Building capacity for the UCD community to engage in patient-centered outcomes research

See below for links to NUCDF's poster presentations.

The satellite symposium offered a forum for the international research community to share cutting-edge research findings and engage in meaningful discussions to push the boundaries of scientific discovery. The full agenda is available here. Scientific presentations were organized into the following themes: 

• Disease Models and Mechanisms – Exploring foundational insights into UCD pathophysiology and novel model systems
• From Phenotypic Diversity to Personalized Medicine – Addressing the clinical variability of UCDs and the move toward individualized treatment approaches
• Innovative vs Established Therapies – Examining emerging treatment strategies in the context of existing standards of care
• Challenges of Newborn Screening for UCDs – Discussing current and evolving practices in early diagnosis

An important feature of the symposium was its focus on the next generation of researchers, offering trainees and junior faculty a platform to present their work, interact with international leaders and build essential connections. Through this experience, the organizers aimed to inspire the next wave of UCD investigators while strengthening global partnerships in the field.

The Citrin Foundation, the European Registry for Intoxication type Metabolic Diseases, and the Japanese Society for Inherited Metabolic Diseases joined NUCDF and UCDC in presenting the symposium. The independent educational program was made possible thanks to educational sponsorships provided by iECURE, Arcturus Therapeutics, and CAMP4 Therapeutics.

NUCDF Poster Presentations

Click to view or download