Introduction
Hyperoxaluria, PH, autosomal recessive genetic disorders, RKSC PH, PH1
PRIMARY HYPEROXALURIA, KIDNEY STONE, HYPEROXALURIA, PRIMARY HYPEROXALURIA
Primary hyperoxaluria (PH) is a family of three rare autosomal recessive genetic disorders–PH1, PH2, PH3–each characterized by a specific deficiency in hepatic glyoxylate metabolism [Previously, PH3 patients have been described as having relatively stable renal function over time, but recent findings from the European Hyperoxaluria Consortium (OxalEurope) Registry and Rare Kidney Stone Consortium Primary Hyperoxaluria (RKSC PH) Registry suggest that this is not always the case [Belostotsky et al. identified mutations in Nedosiran is an RNA interference (RNAi) agent that inhibits hepatic lactate dehydrogenase (LDH) expression (encoded by the The primary objective of this phase I study (PHYOX4) was to evaluate the safety and tolerability of single-dose nedosiran in patients with PH3.