July 17, 2026

Urea Cycle Disorders: The Rhythm of Progress Symposium

Virtual

On this page:

About
Event hosts
Registration
Agenda & CME information

About the conference

Recent advances have expanded research and treatment possibilities for urea cycle and metabolic disorders, yet gaps remain in awareness, diagnosis, and care.

Urea Cycle Disorders: The Rhythm of Progress Symposium, set for Friday, July 17, 2026, convenes leading investigators, clinicians, and translational scientists to examine the current landscape, emerging discoveries, and future directions in urea cycle disorder (UCD) research and care.

This inaugural symposium highlights innovative research, novel diagnostics, and evolving therapeutic strategies. Featuring experts across metabolism, cancer biology, and rare disease therapeutics, this event fosters cross-disciplinary collaboration, training, and mentorship to accelerate translational science and advance meaningful progress for individuals living with UCDs.

Event hosts

Overview

Event: Urea Cycle Disorders: The Rhythm of Progress Symposium

Date: July 17, 2026

Format: Virtual

Program host: Pediatric Translational Neuroscience Initiative

Host:
Andrea Gropman, MD, FAAP, FACMG, FANA, FAAN, FCNS
Pediatric Translational Neuroscience Initiative

Event contact:
PTNI@stjude.org

Registration deadlines & fees

This symposium will be hosted virtually for external participants with limited in-person attendance for internal participants. Registration is free.

Agenda & CME information

Time (Central Time)	Session/Event	Speaker
8:00 a.m.	Meeting link opens
8:30 a.m.–12:15 p.m.	Research Session: Innovations in Urea Cycle Disorders (UCDs) and Rare Disease
8:30–8:40 a.m.	Welcome	Paul Taylor, MD, PhD
8:45–8:50 a.m	Introductions
8:45–9:05 a.m.	A Consortium Approach to Advancing Clinical Research and Clinical Care in UCDs	Sandesh Nagamani, MD
9:05–9:25 a.m.	Noninvasive Biomarkers of Liver Disease in UCDs	Lindsay Burrage, MD
9:25–9:45 a.m.	Deciphering the UCD Brain: Structural, Functional, and Metabolic Signatures of Urea Cycle Dysfunction	Andrea Gropman, MD
9:45–10:05 a.m.	When Two Wrongs Make a Right: Genetic Insights from Yeast Functional Assays	Aimee Dudley, PhD
10:05–10:25 a.m.	Q&A Panel with Speakers 1–4
10:25–10:40 a.m.	Break
10:40–10:45 a.m.	Introductions
10:45–11:30 a.m.	Unraveling the Brain Pathophysiology of Arginase Deficiency	Gerald Lipshutz, MD
11:30 a.m.–12:15 p.m.	Therapeutic Gene Editing for Urea Cycle Disorders	Rebecca Ahrens-Nicklas, MD, PhD
12:15-1:30 p.m.	Break
1:30–4:00 p.m.	Clinical Session: UCD Care and Management
1:30–1:35 p.m.	Introductions
1:35–1:55 p.m.	Neurobehavioral Symptoms of OTCD in Heterozygous Girls and Women.	Greta Wilkening, PhD
1:55–2:25 p.m.	UCD 101	Lindsay Burrage, MD
2:25–2:45 p.m.	Diet Management & Importance of Protein Management	Erin MacLeod, PhD, RD, LD
2:45–3:00 p.m.	Break
3:00–3:05 p.m.	Introductions
3:05–3:35 p.m.	Medical Management in UCD	Laura Konczal, MD
3:35–3:55 p.m.	Cerebral Metabolism in UCDs: Quantifying Changes in MRS and fNIRS	Puneet Bagga, PhD Kosar Khaksari, PhD
3:55–4:00 p.m.	Closing Remarks	Andrea Gropman, MD

Educational Objectives

After participating in this symposium, learners will be able to:

Recognize and diagnose urea cycle disorders across the lifespan, integrating clinical presentation, biochemical testing, and genetic evaluation to reduce time to diagnosis and improve outcomes.
Explain the underlying pathophysiology of urea cycle disorders, including ammonia toxicity, metabolic network disruption, and effects on brain development and function, and apply these concepts to patient management.
Apply evidence-based strategies for acute and long-term management of UCDs, including dietary therapy, nitrogen scavengers, neurocognitive monitoring, and transition of care.
Evaluate new and emerging therapies for urea cycle disorders, including gene-based, enzyme replacement, and precision medicine approaches, and discuss their implications for future clinical practice and research.

Accreditation Statement

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of St. Jude Children’s Research Hospital and the Urea Cycle Disorders Consortium. St. Jude Children’s Research Hospital is accredited by the ACCME to provide continuing medical education for physicians.

Credit Designation Statement

St. Jude Children’s Research Hospital designates this live activity for a maximum of 4 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

About the St. Jude Pediatric Translational Neuroscience Initiative (PTNI)

St. Jude is committed to transforming the treatment landscape for children living with catastrophic pediatric neurological disorders through PTNI. Launched in 2020, is a formalized mechanism for translational neuroscience that extends from fundamental bench science to experimental neurotherapeutics with an emphasis on working closely with stakeholders across multiple sectors, including other researchers and institutions, patient and community groups, industry, and policymakers.

Through collaborations and thought partnership, PTNI engages thought leaders to identify promising paths forward in the pediatric neuroscience space, convening scientists, clinicians, patient advocates, and others to address the unmet needs of children with these disorders.

Learn more about PTNI