Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3

Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3

Introduction: Primary hyperoxaluria (PH) is a rare genetic disorder of hepatic glyoxylate metabolism. Nedosiran is an RNA interference (RNAi) therapeutic that the US Food and Drug Administration has approved for treatment of PH1. PHYOX3 is a trial evaluating monthly nedosiran in patients with PH. Me...

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Main Authors: Jaap Groothoff, Anne-Laure Sellier-Leclerc, Lisa Deesker, Justine Bacchetta, Gesa Schalk, Burkhard Tönshoff, Graham Lipkin, Sandrine Lemoine, Thomas Bowman, Jing Zhou, Bernd Hoppe
Format: Article
Language:English
Published: Elsevier 2024-05-01
Series:Kidney International Reports
Subjects:
glyoxylate metabolism
nedosiran
primary hyperoxaluria type 1
RNAi therapy
safety/efficacy
urinary oxalate excretion
Online Access:http://www.sciencedirect.com/science/article/pii/S2468024924015298
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