Today, on International Arginase 1 Deficiency (ARG1-D) Day, we join families, patients, clinicians, researchers, and advocates around the world to raise awareness of this ultra-rare disease and celebrate a year of extraordinary progress.

For decades, families affected by ARG1-D faced a future focused primarily on managing symptoms, slowing progression, and preserving mobility and cognitive function for as long as possible. This year marks a historic turning point.

In February 2026, Immedica Pharma, headquartered in Stockholm, Sweden, received U.S. FDA approval for LOARGYS® (pegzilarginase), the first and only approved therapy specifically for Arginase 1 Deficiency. This milestone represents years of dedication by patients, families, researchers, clinicians, medical providers, advocacy organizations, and industry partners working together toward a common goal. LOARGYS offers a new treatment option that addresses the underlying enzyme deficiency and has brought renewed optimism to the global ARG1-D community.

While we celebrate this achievement, our work is far from finished. We remain committed to improving diagnosis, expanding newborn screening, supporting research, connecting families worldwide, and ensuring every individual living with ARG1-D has access to the care and resources they need.

The Arginase 1 Deficiency Foundation is also proud to continue our partnership with the National Urea Cycle Disorders Foundation (NUCDF). Together, we will bring families together at the 2026 Family Conference in Memphis, July 17–19, where patients, caregivers, clinicians, and researchers will gather to learn, connect, and strengthen our community.

International ARG1-D Day is a reminder that progress is possible when families refuse to give up, researchers continue to push forward, and a community stands together. Every diagnosis matters. Every family matters. Every step forward matters.

Today we celebrate resilience, progress, and the brighter future being created for everyone living with ARG1-D.