From June 9th-11th, the Foundation hosted its third in-person symposium on citrin deficiency at the Møller Institute, University of Cambridge. Spanning two and a half days, the scientific program featured a dynamic schedule of talks and a dedicated poster session. Topics ranged from exploring the biochemical mechanisms, new preclinical models, new diagnostic and bioprofiling tools to advancements in therapeutic approaches for citrin deficiency.
On the final day, the sessions explored topics ranging from clinical trial design and execution to the financial viability of novel therapies for rare diseases and AI-driven drug repurposing. Six posters from the poster walk had also been selected for flash talk presentations, offering researchers a platform to showcase their work. In addition to the formal program, the Foundation hosted an afternoon of social activities, giving attendees an opportunity to experience Cambridge while continuing to build relationships in a more informal setting.
This was our largest symposium to date, reflecting both the momentum of ongoing research and the continued growth of our community through expanded funding and an increasingly connected consortium network. More than 80 delegates (clinicians, scientists, dietitians, biotech representatives, patients and families) joined us from Australia, Bulgaria, Canada, Finland, Italy, Japan, Malaysia, Singapore, South Korea, Spain, Sweden, Switzerland, Taiwan, the United Kingdom, the United States, and Vietnam.
While citrin deficiency remained the central focus, the event also addressed broader rare disease challenges through cross-sector collaboration. We move forward with renewed conviction that this gathering has helped accelerate our shared mission—and we are excited for what lies ahead.
Thank you again to all who participated and contributed to this remarkable event. To relive some highlights, we invite you to visit our YouTube channel and view the symposium highlights reel from below.
