International ARG1-D Day – June 10, 2026
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 […]
Historic News For The Arg1-d Community
Yesterday, the U.S. Food and Drug Administration (FDA) approved Pegzilarginase (Loargys) — the first and only treatment for Arginase 1 Deficiency (ARG1-D). This is not just a milestone. This is hope realized. For families living with ARG1-D, this approval represents something we have waited for, worked toward, advocated for, and prayed over for years — […]
U.S. FDA has granted accelerated approval of Loargys®
Dear Arginase 1 Deficiency Community & Families, If you have not seen already, we are excited to share that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Loargys® (pegzilarginase-nbln), a prescription medicine used to treat high levels of arginine in the blood of adults and children 2 years and older with […]
Thank You for Making Hope on the Court Unforgettable
Dear Friends, Sponsors, and Hope on the Court Community,
From the bottom of our hearts, thank you for making the Hope on the Court Pickleball Tournament such an extraordinary and meaningful day. Because of you, this event was far more than a tournament—it was a celebration of connection, generosity, and purpose.
Immedica Publishes Favorable Long-Term Data on Loargys® (pegzilarginase)
Stockholm, August 29, 2025 – Immedica is pleased to announce the publication of favorable long-term data on Loargys (pegzilarginase), demonstrating sustained clinical benefits including clinically significant improvements in mobility and spasticity in patients 2 years and older with Arginase 1 deficiency (ARG1-D). ARG1-D is an autosomal recessive urea cycle disorder (UCD) characterized by chronic hyperargininemia, leading […]
NUCDF Awards Cynthia Le Mons Fellowship to Molecular Biologist for Arginase-1 Deficiency Research
The National Urea Cycle Disorders Foundation (NUCDF) is pleased to announce that it has awarded the 2025 Cynthia Le Mons Fellowship to Shradha Suyal, PhD, a postdoctoral fellow at the Pacific Northwest Research Institute (PNRI). Suyal (pictured above, left) works in the lab of Aimée Dudley, PhD (pictured above, right), who uses yeast genetics to study the severity […]
Stephen Cederbaum honored with lifetime achievement award in medical genetics
Dr. Stephen Cederbaum, a longtime UCLA Health physician and renowned expert in genetic medicine, has been awarded the 2025 David L. Rimoin Lifetime Achievement Award by the ACMG Foundation for Genetic and Genomic Medicine. The award honors professionals who have dedicated their career to advancing science and improving lives.
BLA for pegzilarginase in the treatment of arginase 1 deficiency (ARG1-D) accepted for priority review by the U.S. FDA
Stockholm, November 5, 2024: Immedica announces today that the Biologics License Application (BLA) for pegzilarginase in the treatment of arginase 1 deficiency (ARG1-D) has been successfully validated and accepted for priority review by the U.S. Food and Drug Administration (FDA). Anders Edvell, CEO of Immedica commented: “We are pleased that the FDA has accepted the […]
Immedica initiates a phase 3 pediatric study for Loargys® (pegzilarginase) in arginase 1 deficiency
Loargys is approved in the EU and Great Britain for the treatment of ARG1-D in adults, adolescents and children aged 2 years and older. The initiation of a clinical study in children below 2 years of age is of high importance in this progressive disease exposing patients to elevated toxic levels of arginine from birth.
Immedica presents new data highlighting treatment benefits of Loargys® (pegzilarginase) in arginase 1 deficiency at the SSIEM congress
Stockholm, September 3, 2024: Immedica announces today that new scientific data on Loargys® (pegzilarginase), the first disease modifying treatment in arginase 1 deficiency, has been accepted at the 2024 Annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Porto, Portugal on 3-6 September.