RNA interference is a naturally occurring cellular mechanism for regulating gene expression mediated by small interfering RNAs. Recent data suggest that this may be a beneficial approach for reducing oxalate production in patients with PH1. The RNA inhibitor lumasiran was approved in 2020 for PH1.
Early studies have suggested pluripotent stem cell-based therapies may be beneficial in PH1. Enzyme restoration therapy, gene editing, and inflammasome inhibitors have shown evidence of efficacy in preclinical studies, but to date, no compounds are under investigation in humans.
Learn more about the diagnosis and management of PH1.
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Cite this: Bradley Schwartz. Fast Five Quiz: Primary Hyperoxaluria Type 1 Management - Medscape - Mar 04, 2022.