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 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 [...]
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), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. The approval follows the positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human [...]
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 at UCLA have received more than $16 million in grants from the California Institute for Regenerative Medicine, the state’s stem cell agency, to [...]
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. While born with normal function, by the age of two, patients with the condition have progressive deterioration in their physical ability due to the gradual accumulation of arginine and ammonia [...]
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 opinion recommending marketing authorization of Loargys® (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D) in patients two years and older. The positive opinion from the CHMP is now referred [...]
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.