Arginase 1 Deficiency News
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. [...]
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® [...]
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.
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 [...]
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 [...]
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.
Massachusetts man with rare disease denied experimental drug treatment
"It's been a long one," Alexandra Eaton told WBZ-TV from her Marshfield home. "It's been challenging, but he's persevered." You can see the love in Eaton's eyes, looking at her son, whose care and comfort consumes her entire life. Josh, who is 26, lives with rare and debilitating Arginase 1 Deficiency.
Loargys® (pegzilarginase) approved in the EU for treatment of arginase 1 deficiency (ARG1-D)
Stockholm, December 18, 2023: Immedica today announces that the European Commission has granted marketing authorization of Loargys® (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents [...]
UCLA scientists receive $16.1 million in CIRM grants to advance novel stem-cell based treatments
From left to right: Dr. Gerald Lipshutz, Dr. Zulema Romero Garcia and Dr. Donald Kohn Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research [...]