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Stephanie Perez Kerkvliet and Michelle N. Rheault

Primary hyperoxaluria type 1 (PH1) is a rare metabolic disease that leads to oxalate overproduction and results in kidney stones, nephrocalcinosis, kidney failure, and eventually systemic oxalosis. Garrelfs et al. (1) recently published results of the multinational, randomized, double-blind, placebo-controlled ILLUMINATE-A clinical trial evaluating the effectiveness of treating PH1 with lumasiran, an RNA interference (RNAi) agent directed against the mRNA encoding glycolate oxidase in the liver. The trial included 39 participants with PH1, ages 6-60 years (median age 14 years, including 22 pediatric participants), with an estimated glomerular filtration rate (eGFR) greater than or equal to 30 mL/min/1.73