Efficacy and Safety of Lumasiran for Advanced Primary Hyperoxaluria Type 1: 24-Month Follow-Up of the Phase 3 Illuminate-C Trial.
(2025) American Journal of Kidney Diseases — Vol. 86, n° 2, p. 285-288 (2025)
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Sellier-LeclercetalEfficacyandSafetyofLumasiranforAdvancedPrimaryHyperoxaluriaType124-monthyfollow-upofthePhas.pdf
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- (en) TO THE EDITOR : Primary hyperoxaluria type 1 (PH1) is a rare autosomal recessive disorder caused by hepatic alanine-glyoxylate aminotransferase deficiency, leading to oxalate overproduction, which results in progressive kidney damage and increased urinary oxalate (UOx) and plasma oxalate (POx) levels. Oxalate accumulation can result in kidney stones, nephrocalcinosis, and kidney failure. In chronic kidney disease stages 3b-5, elevated POx increases the risk of calcium oxalate deposition (systemic oxalosis) in the heart, bones, eyes, and other organs. [...]
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Sellier-Leclerc, A.-L., Magen, D., Shasha-Lavsky, H., Simkova, E., Devresse, A., Guebre-Egziabher, F., Michael, M., Lieske, J. C., Frishberg, Y., Bakkaloglu, S. A., Mourani, C., Saqan, R., Singer, R., Guzzo, I., Makarova, N., Willey, R., Kaspar, C., Gansner, J. M., & Groothoff, J. W. (2025). Efficacy and Safety of Lumasiran for Advanced Primary Hyperoxaluria Type 1: 24-Month Follow-Up of the Phase 3 Illuminate-C Trial. American Journal of Kidney Diseases, 86(2), 285-288. https://doi.org/10.1053/j.ajkd.2025.01.016 (Original work published 2025)