Results 1 to 10 of about 131 (70)

Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency. [PDF]

J Inherit Metab Dis, 2021
Hyperargininemia in patients with arginase 1 deficiency (ARG1- D) is considered a key driver of disease manifestations, including spasticity, developmental delay, and seizures. Pegzilarginase (AEB1102) is an investigational enzyme therapy which is being
Diaz GA, Schulze A, McNutt MC, Leão-Teles E, Merritt JL, Enns GM, Batzios S, Bannick A, Zori RT, Sloan LS, Potts SL, Bubb G, Quinn AG.   +12 more
europepmc   +7 more sources

Pegzilarginase in Arginase 1 Deficiency: Clinical and Biochemical Effects of Treatment Initiation, Discontinuation and Re-Initiation [PDF]

Children
Background: Arginase 1 deficiency (ARG1-D) is an ultra-rare urea cycle disorder characterized by hyperargininemia and progressive neurological impairment, including spasticity, loss of motor function, and reduced quality of life.
Martha Caterina Faraguna, Viola Crescitelli, Roberta Pretese, Maria Valvassori Bolgè, Vera Marchetti, Giusi Sgroi, Stefania Sala, Silvia Gigante, Cristina Bonfanti, Adriana Balduzzi, Serena Gasperini   +10 more
doaj   +5 more sources

Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports [PDF]

JIMD Reports, 2022
Background Arginase 1 deficiency (ARG1‐D) is a rare, progressive and debilitating urea cycle disorder characterized by clinical manifestations including spasticity, seizures, developmental delay, and intellectual disability.
Aseel Bin Sawad, Arti Pothukuchy, Mark Badeaux, Victoria Hodson, Gillian Bubb, Kristina Lindsley, Jennifer Uyei, George A. Diaz   +7 more
doaj   +3 more sources

Arginine Deprivation in SCLC: Mechanisms and Perspectives for Therapy [PDF]

Lung Cancer: Targets and Therapy, 2022
Joséphine Carpentier,1 Iuliia Pavlyk,1 Uma Mukherjee,2 Peter E Hall,2 Peter W Szlosarek1,2 1Centre for Cancer Biomarkers and Biotherapeutics, Barts Cancer Institute, Queen Mary University of London, London, EC1M 6BQ, UK; 2Department of Medical Oncology ...
Carpentier J, Pavlyk I, Mukherjee U, Hall PE, Szlosarek PW   +4 more
doaj   +4 more sources

Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trialResearch in context [PDF]

EClinicalMedicine
Summary: Background: Arginase 1 Deficiency (ARG1-D) is a rare debilitating, progressive, inherited, metabolic disease characterized by marked increases in plasma arginine (pArg) and its metabolites, with increased morbidity, substantial reductions in ...
Rossana Sanchez Russo, Serena Gasperini, Gillian Bubb, Linda Neuman, Leslie S. Sloan, George A. Diaz, Gregory M. Enns   +6 more
doaj   +5 more sources

Retrospective analysis of arginase 1 deficiency progression in adults over 5 years at a single metabolic centre [PDF]

JIMD Reports
Background The clinical presentation of ARG1‐D is characterised by elevated arginine levels leading to neurological and mobility impairments. Information about long‐term outcomes in adults is lacking, which prompted us to undertake a retrospective ...
Reena Sharma, John Bassett, Karolina M. Stepien, Andrew Oldham, Ana Jovanovic, Alison Woodall, Diane Green   +6 more
doaj   +3 more sources

O08: Long-term efficacy and safety of pegzilarginase for arginase 1 deficiency: 2 years of experience in the phase 2 extension study*

Genetics in Medicine Open, 2023
Markey McNutt, George Diaz, Andreas Schulze, Gillian Bubb, Leslie Sloan, Elisa Leão-Teles, Spyros Batzios, Angela Sun, Allison Bannick, Roberto Zori, Gregory Enns   +10 more
doaj   +3 more sources

Arginase 1 deficiency: a treatable form of spastic paraplegia. [PDF]

Neurol Sci
Burlina A, Ardissone A, Battini R, Burlina A, Gasperini S, Pession A, Porta F, Vici CD.   +7 more
europepmc   +2 more sources

Arginase therapy combines effectively with checkpoint blockade or agonist anti-OX40 immunotherapy to control tumor growth.

, 2021
Metabolic dysregulation is a hallmark of cancer; for instance, many tumors exhibit auxotrophy for various amino acids, such as arginine, as they are unable to meet the demand for these amino acids through endogenous production.
Van Cleef, Jessica, Priddy, Leslie, Rajamanickam, Venkatesh, Agnello, Giulia, Enzler, Danlee, Kasiewicz, Melissa, Rolig, Annah S, Vander Heiden, Matthew G, Redmond, William L, Badeaux, Mark D, Daige, Christopher, Sullivan, Mark R, Wooldridge, James, Muir, Alexander, Wiggins, Jason F, Rowlinson, Scott W   +15 more
core   +1 more source

Pegzilarginase in combination with agonist anti-OX40 therapy enhances T cell priming and effector function leading to improved tumor regression and survival

, 2018
Background: Tumor cells defective in enzymes required for arginine biosynthesis are dependent upon arginine uptake from the environment. Extracellular depletion of arginine directly affects tumor cells, inducing autophagy and apoptosis.
Badeaux, Mark, Rowlinson, Scott, Sturgill, Elizabeth, Rolig, Annah, Kasiewicz, Melissa, Redmond, William L.   +5 more
core   +2 more sources