Journal article
Nedosiran in pediatric patients with PH1 and relatively preserved kidney function, a phase 2 study (PHYOX8)
Abstract
BackgroundPrimary hyperoxaluria type 1 (PH1) is an autosomal recessive disorder with dysregulated glyoxylate metabolism in the liver. Oxalate over-production leads to renal stones, progressive kidney damage and renal failure, with potentially life-threatening systemic oxalosis. Nedosiran is a synthetic RNA interference therapy, designed to reduce hepatic lactate dehydrogenase (LDH) to decrease oxalate burden in PH.MethodsCurrently, in the …
Authors
Sas DJ; Bakkaloglu SA; Belostotsky V; Hayes W; Ariceta G; Zhou J; Rawson V
Journal
Pediatric Nephrology, Vol. 40, No. 6, pp. 1939–1948
Publisher
Springer Nature
Publication Date
June 2025
DOI
10.1007/s00467-025-06675-8
ISSN
0931-041X