On behalf of the Organizing Committee, Symposium Co-Chairs Andrea Gropman (Chief of Neurogenetics at Children’s National Hospital in Washington, DC, Co-PI of the NIH Rare Diseases Clinical Research Network Urea Cycle Disorders Consortium) and Cynthia Le Mons (Executive Director, National Urea Cycle Disorders Foundation, Co-PI of the NIH Rare Diseases Clinical Research Network Urea Cycle Disorders Consortium) welcomed over 200 international participants to the 5th International Urea Cycle Disorders Satellite Symposium, “Mapping Progress: Current Concepts and New Insights in Urea Cycle Disorders,” a satellite of the 14th International Congress on Inborn Errors of Metabolism held in Sydney, Australia. The Symposium continued a tradition begun in 1997 with the first such Symposium held as a satellite of the 7th International Congress on Inborn Errors of Metabolism in Vienna, followed in 2003 in Sydney, 2009 in La Jolla, and 2013 in Barcelona. In each case, the proceedings were published and served as a permanent record of the state of the art in these disorders and a benchmark against which our current understanding of UCD and their treatment modalities can be measured. Accordingly, the proceedings of this Symposium will be published as a special supplement in Molecular Genetics and Metabolism.
The purpose of this meeting was to once again bring together experts in the field of urea cycle disorders for an update on the current understanding of UCD treatment and the different ways we view the field, and to share new insights. We have made exciting progress, and by embracing a culture of collaboration and new technologies, together we can make transformational progress.
This year’s meeting differed in some ways from its predecessors. We’ve all faced challenges with the COVID-19 pandemic, and this was our first “virtual” symposium. With our state-of-the-art virtual platform, participants were able to access all the presentations, participate in live Q&A, attend our poster session, access abstracts and network with colleagues.
Our program focused on those elements and issues in UCD where there is universal concern across the US and the world, focusing on best practices in clinical care. Molecular definition, the creation of animal models and aspects of the physiology have been discussed in previous symposia. The neuroscience session of our program is meant to stimulate essential discussions about neuroprotection and its role in preventing the devastating effects of UCD on brain function. Our sessions on brain, liver, and our FDA session on biomarkers are meant to encourage discussion about how our research findings and noninvasive monitoring can be used in clinical trials. Our speakers represent all the corners of the globe and speak to the international diversity of the field.
The hope is that this symposium will be a prologue to revolutionary progress in the treatment and understanding of UCDs that sets the stage for our next satellite in 2027 at the 15th International Congress on Inborn Errors of Metabolism in Japan.
