Lessons for the clinical nephrologist: lumasiran as the future cornerstone treatment for patients with primary hyperoxaluria type 1?

Gillion, Valentine;Dahan, Karin;Scohy, Anaïs;Devresse, Arnaud;Godefroid, Nathalie
(2023) Journal of Nephrology — Vol. 36, n° 2, p. 329-333 (2023)

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Abstract
(en) Primary hyperoxaluria type 1 (PH1)—OMIM #259900—is a rare recessive autosomal disorder caused by a deficiency of the liver peroxisomal enzyme alanine-glyoxylate-aminotransferase (AGT), which catalyzes the conversion of glyoxylate to glycine. Reduced AGT activity leads to the conversion of glyoxylate to oxalate (Fig. 1). Oxalate forms insoluble calcium oxalate crystals that accumulate in the kidney and subsequently in other organs, when the kidneys are saturated, leading to systemic oxalosis. The most severe PH1 cases start during the first months of life with rapid development of kidney failure. [...]
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Gillion, V., Dahan, K., Scohy, A., Devresse, A., & Godefroid, N. (2023). Lessons for the clinical nephrologist: lumasiran as the future cornerstone treatment for patients with primary hyperoxaluria type 1? Journal of Nephrology, 36(2), 329-333. https://doi.org/10.1007/s40620-022-01435-5 (Original work published 2023)