Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Garrelfs, SF; Frishberg, Y; Hulton, SA; Koren, MJ; O'Riordan, WD; Cochat, P; Deschenes, G; Shasha-Lavsky, H; Saland, JM; van't Hoff, WG; Fuster, DG; Magen, D; Moochhala, SH; Schalk, G; Simkova, E; Groothoff, JW; Sas, DJ; Meliambro, KA; Lu, J; Sweetser, MT; Garg, PP; Vaishnaw, AK; Gansner, JM; McGregor, TL; Lieske, JC

Garrelfs, SF (corresponding author), Univ Amsterdam, Amsterdam UMC, Emma Childrens Hosp, Dept Pediat Nephrol,Locat AMC, Rm H8-253,Meibergdreef 9, NL-1105 AZ Amsterdam, Noord Holland, Netherlands.

NEW ENGLAND JOURNAL OF MEDICINE, 2021; 384 (13): 1216

Abstract

BackgroundPrimary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate that leads to kidney stones, nephro......

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