Results 61 to 70 of about 131 (70)

Phase 1 trial of pegzilarginase in patients (pts) with relapsed/refractory (R/R) AML or MDS refractory to hypomethylating agents (HMAs).

Journal of Clinical Oncology, 2018
7031Background: In vitro studies demonstrate that AML cells are arginine auxotrophs and are metabolically vulnerable to arginine depletion (PMID: 24018014 & 25896651).
Geoffrey L. Uy, Michael R. Savona, Benjamin K. Tomlinson, Hetty Carraway, Dale L. Bixby, Sarit E. Assouline, Joseph M. Brandwein, Moshe Yair Levy, Robert Collins, Humberto Lara-Guerra, Susan Alters, Stephen Eckert, Scott Rowlinson, James Wooldridge, Aaron David Schimmer   +14 more
openaire   +1 more source

Pegzilarginase and beyond: Italian real-world experience and proposal of innovative tools for comprehensive management of ARG1-D

Journal of Innate Metabolism
Arginase 1 deficiency (ARG1-D) is a rare urea cycle disorder primarily affecting the central nervous system with progressive spasticity, cognitive decline, and variable seizures, despite often mild hyperammonemia. Conventional treatment fails to maintain optimal metabolic control, leading to irreversible functional impairment.
Gasperini, S., Martinelli, D., Porta, F.
openaire   +1 more source

PEGZILARGINASE IN ARGINASE 1 DEFICIENCY: RESULTS OF THE PEACE PIVOTAL PHASE 3 CLINICAL TRIAL

Molecular Genetics and Metabolism, 2022
Gregory M. Enns, Rossana Sanchez Russo, Eric Bradford, Gillian Bubb, Leslie S. Sloan, Serena Gasperini, George A. Diaz   +6 more
openaire   +1 more source

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Case Medical Research, 2019
openaire   +1 more source

Clinical effects of pegzilarginase in patients with arginase-1 deficiency

Molecular Genetics and Metabolism
Neil Jacob, Alyne Restrepo, Mary Freeman, Louise Bier, Brandon Stauffer, Chunli Yu, Jaya Ganesh   +6 more
openaire   +1 more source

Pegzilarginase for treatment of hyperargininemia in arginase 1 deficiency: FDA approval summary

Molecular Genetics and Metabolism
Jennifer Evelyn Shields, Yared Gurmu
openaire   +1 more source

LOARGYS (pegzilarginase-nbln)

Clinical Therapeutics
openaire   +2 more sources

Correction to “Long‐Term Efficacy and Tolerability of Pegzilarginase in Arginase 1 Deficiency: Results of Two International Multicentre Open‐Label Extension Studies”

Journal of Inherited Metabolic Disease
openaire   +1 more source

Pegzilarginase (Hyperargininämie); Bewertung gemäß § 35a Abs. 1 Satz 11 SGB V

openaire   +1 more source