Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Primary hyperoxaluria type 1 is caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested whether an oligonucleotide drug can reduce the production of hepatic oxalate.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2021-04, Vol.384 (13), p.1216-1226
Hauptverfasser:	Garrelfs, Sander F, Frishberg, Yaacov, Hulton, Sally A, Koren, Michael J, O’Riordan, William D, Cochat, Pierre, Deschênes, Georges, Shasha-Lavsky, Hadas, Saland, Jeffrey M, van’t Hoff, William G, Fuster, Daniel G, Magen, Daniella, Moochhala, Shabbir H, Schalk, Gesa, Simkova, Eva, Groothoff, Jaap W, Sas, David J, Meliambro, Kristin A, Lu, Jiandong, Sweetser, Marianne T, Garg, Pushkal P, Vaishnaw, Akshay K, Gansner, John M, McGregor, Tracy L, Lieske, John C
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Age Calcinosis Child Creatinine Creatinine - urine Double-Blind Method Enzymes Excretion Female Glomerular Filtration Rate Humans Hyperoxaluria Hyperoxaluria, Primary - blood Hyperoxaluria, Primary - complications Hyperoxaluria, Primary - drug therapy Hyperoxaluria, Primary - urine Kidney Calculi - prevention & control Kidney diseases Kidney stones Laboratories Liver Liver diseases Male Metabolism Middle Aged Nephrolithiasis Ostomy Oxalates - blood Oxalates - metabolism Oxalates - urine Oxalic acid Oxalosis Placebos Plasma Primary hyperoxaluria Renal failure RNA, Small Interfering - adverse effects RNA, Small Interfering - therapeutic use RNA-mediated interference RNAi Therapeutics Statistical analysis Young Adult
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Beschreibung
Zusammenfassung:	Primary hyperoxaluria type 1 is caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested whether an oligonucleotide drug can reduce the production of hepatic oxalate.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2021712