1. Academic Validation
  2. Nedosiran Dramatically Reduces Serum Oxalate in Dialysis-Dependent Primary Hyperoxaluria 1: A Compassionate Use Case Report

Nedosiran Dramatically Reduces Serum Oxalate in Dialysis-Dependent Primary Hyperoxaluria 1: A Compassionate Use Case Report

  • Urology. 2021 Oct;156:e147-e149. doi: 10.1016/j.urology.2021.03.014.
Kevin Shee 1 Justin Ahn 2 Fadl Hamouche 2 Jorge Mena 2 Thomas Chi 2 Marshall L Stoller 3
Affiliations

Affiliations

  • 1 Department of Urology, UCSF, San Francisco, CA. Electronic address: kevin.shee@ucsf.edu.
  • 2 Department of Urology, UCSF, San Francisco, CA.
  • 3 Department of Urology, UCSF, San Francisco, CA. Electronic address: marshall.stoller@ucsf.edu.
Abstract

Primary hyperoxaluria 1 (PH1) is a devastating condition involving recurrent urolithiasis, early end-stage renal disease and multisystemic deposition of calcium oxalate crystals. Treatment options for PH1 are limited, inevitably requiring transplantation, usually combined kidney and liver transplant. Here we report successful compassionate use of Nedosiran, an RNA interference targeting Lactate Dehydrogenase, in an index patient. Monthly Nedosiran injections led to dramatically decreased plasma oxalate levels, decreased frequency of weekly hemodialysis sessions from 6 to 3, and deferral of combined kidney and liver transplant. Nedosiran represents a novel and impactful potential therapeutic for PH1 patients with end-stage renal disease.

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