Efficacy and safety of pegzilarginase in patients below 2 years of age with arginase 1 deficiency: a phase 3, open-label, multi-centre study. [PDF]
EClinicalMedicineArginase 1 Deficiency (ARG1-D) is a rare metabolic disorder characterized by marked hyperargininaemia and progressive neurological impairment. Dietary protein restriction alone is often insufficient to normalize plasma arginine (pArg) or prevent disease progression.
Ghosh A +3 more
europepmc +3 more sources
A Phase 1 study of ADI-PEG20 (pegargiminase) combined with cisplatin and pemetrexed in ASS1-negative metastatic uveal melanoma. [PDF]
Pigment Cell Melanoma Res, 2022 Abstract Metastatic uveal melanoma (UM) is a devastating disease with few treatment options. We evaluated the safety, tolerability and preliminary activity of arginine depletion using pegylated arginine deiminase (ADI‐PEG20; pegargiminase) combined with pemetrexed (Pem) and cisplatin (Cis) chemotherapy in a phase 1 dose‐expansion study of patients with
Chan PY +11 more
europepmc +2 more sources
Cancer Research, 2018 Abstract Tumors unable to synthesize L-Arginine (arginine) due to decreased expression of enzymes of the arginine biosynthetic pathway show increased sensitivity to arginine depletion. Pegzilarginase (AEB1102) is a bioengineered human PEGylated arginase 1 with enhanced pharmacological properties that enables marked depletion of arginine ...
Diwakar Davar +9 more
core +2 more sources
American Journal of Medical Genetics Part A, Volume 185, Issue 11, Page 3350-3358, November 2021., 2021 Abstract From Sir Archibald Garrod's initial description of the tetrad of albinism, alkaptonuria, cystinuria, and pentosuria to today, the field of medicine dedicated to inborn errors of metabolism has evolved from disease identification and mechanistic discovery to the development of therapies designed to subvert biochemical defects.
Hilary J. Vernon, Irini Manoli
wiley +1 more source
Novel drugs approved by the EMA, the FDA, and the MHRA in 2023: A year in review
British Journal of Pharmacology, Volume 181, Issue 11, Page 1553-1575, June 2024.Abstract In 2023, seventy novel drugs received market authorization for the first time in either Europe (by the EMA and the MHRA) or in the United States (by the FDA). Confirming a steady recent trend, more than half of these drugs target rare diseases or intractable forms of cancer.
Andreas Papapetropoulos +16 more
wiley +1 more source
Challenges and opportunities in the development of combination immunotherapy with OX40 agonists. [PDF]
Expert Opin Biol Ther, 2023 Redmond WL.
europepmc +1 more source
Human arginase 1, a Jack of all trades? [PDF]
3 Biotech, 2022 Anakha J +5 more
europepmc +1 more source
Targeting Cancer Metabolism to Improve Outcomes with Immune Checkpoint Inhibitors. [PDF]
J Immunother Precis Oncol, 2023 Fatima Z, Abonofal A, Stephen B.
europepmc +1 more source
Arginine depletion as a therapeutic approach for patients with COVID-19. [PDF]
Int J Infect Dis, 2021 Grimes JM +5 more
europepmc +1 more source
Human arginase I: a potential broad-spectrum anti-cancer agent. [PDF]
3 Biotech, 2023 Anakha J, Prasad YR, Sharma N, Pande AH.
europepmc +1 more source