Journal
NATURE REVIEWS UROLOGY
Volume 19, Issue 3, Pages 137-146Publisher
NATURE PORTFOLIO
DOI: 10.1038/s41585-021-00543-4
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Primary hyperoxalurias are a devastating family of diseases that eventually lead to end-stage renal disease. Current treatment paradigms are limited, but new therapeutics aim to target oxalate synthesis and metabolism pathways to reduce accumulation and deposition in the body.
Primary hyperoxalurias are a devastating family of diseases leading to multisystem oxalate deposition, nephrolithiasis, nephrocalcinosis and end-stage renal disease. Traditional treatment paradigms are limited to conservative management, dialysis and combined transplantation of the kidney and liver, of which the liver is the primary source of oxalate production. However, transplantation is associated with many potential complications, including operative risks, graft rejection, post-transplant organ failure, as well as lifelong immunosuppressive medications and their adverse effects. New therapeutics being developed for primary hyperoxalurias take advantage of biochemical knowledge about oxalate synthesis and metabolism, and seek to specifically target these pathways with the goal of decreasing the accumulation and deposition of oxalate in the body. Primary hyperoxalurias are a devastating family of diseases that eventually lead to end-stage renal disease. In this Review, Shee and Stoller discuss current treatment paradigms for primary hyperoxalurias, new therapeutics and their mechanisms of action, and future directions for novel research in the field.
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