News & Info

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

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

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

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.

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

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

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.

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

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.

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.

“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.

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),

Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received more than $16 million in grants

By Heather McKenzie See Orignial Story Here Arginase 1 deficiency, or ARG1-D, is a rare, inherited disorder occurring in approximately 1.1 in 1 million live U.S. births.

Stockholm, October 13, 2023: Immedica today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive

The FDA wouldnʼt review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.

Aeglea BioTherapeutics Announces Sale of Pegzilarginase to Immedica Pharma Global rights to pegzilarginase in development for Arginase 1 Deficiency sold to Immedica Pharma for $15

Arginase 1 Deficiency Foundation Files Petition Urging Aeglea Biotherapeutics to Reinstate Life-Saving Drug Following Abrupt Withdrawal to Clinical Trial Patients in the United States Seattle,

We’re proud to partner with the Child Neurology Foundation to conduct a survey to understand caregivers’ experiences on their journey in child neurology. Results will

PEGZILARGINASE Trial Discussion. Amazing presentation with Dr. Diaz, Dr. Burton and our own Tanja Brandt discuss Arginase 1 Deficiency, the Phase 2 and Phase 3

I am excited to share with you that today we announced our Phase 3 PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) study achieved

On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with officials

Aeglea BioTherapeutics and Immedica Announce Commercialization Agreement for Pegzilarginase for the Treatment of Arginase 1 Deficiency in Europe and Middle East Published: Mar 22, 2021

Aeglea BioTherapeutics Launches THINK ARGININE™, a Disease Education Initiative to Improve Awareness and Diagnosis of Arginase 1 Deficiency 05/06/2021 Original Story HERE Includes Sponsored Diagnostic