The effect of lumasiran therapy for primary hyperoxaluria type 1 in small infants

Meaux, MN; Sellier-Leclerc, AL; Acquaviva-Bourdain, C; Harambat, J; Allard, L; Bacchetta, J

Bacchetta, J (通讯作者)，Hosp Civils Lyon, Serv Nephrol Rhumatol & Dermatol Pediat, Ctr Reference Malad Renales Rares Nephrogones Fil, Bron, France.;Bacchetta, J (通讯作者)，Hosp Civils Lyon, Serv Biochim & Biol Mol Malad Hereditaires Metab, Bron, France.;Bacchetta, J (通讯作者)，Univ Claude Bernard, Fac Med Lyon Est, Lyon, France.

PEDIATRIC NEPHROLOGY, 2022; 37 (4): 907

Abstract

Background Lumasiran, a sub-cutaneous RNA-interference therapy, has been recently approved for primary hyperoxaluria type 1 (PH1), with doses and inte......

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