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Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

by Garrelfs, Sander F , Groothoff, Jaap W , McGregor, Tracy L , Magen, Daniella , O’Riordan, William D , Deschênes, Georges , Gansner, John M , Lieske, John C , Moochhala, Shabbir H , Cochat, Pierre , Saland, Jeffrey M , van’t Hoff, William G , Lu, Jiandong , Koren, Michael J , Sas, David J , Shasha-Lavsky, Hadas , Vaishnaw, Akshay K , Sweetser, Marianne T , Fuster, Daniel G , Schalk, Gesa , Hulton, Sally A , Meliambro, Kristin A , Frishberg, Yaacov , Garg, Pushkal P , Simkova, Eva

in 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 / Life Sciences / Liver / Liver diseases / Male / Metabolism / Middle Aged / Nephrolithiasis / Ostomy / Other / 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

2021

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Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

2021

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Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

2021

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Journal Article

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Garrelfs, Sander F,

Groothoff, Jaap W,

McGregor, Tracy L,

Magen, Daniella,

O’Riordan, William D,

Deschênes, Georges,

Gansner, John M,

Lieske, John C,

Moochhala, Shabbir H,

Cochat, Pierre,

Saland, Jeffrey M,

van’t Hoff, William G,

Lu, Jiandong,

Koren, Michael J,

Sas, David J,

Shasha-Lavsky, Hadas,

Vaishnaw, Akshay K,

Sweetser, Marianne T,

Fuster, Daniel G,

Schalk, Gesa,

Hulton, Sally A,

Meliambro, Kristin A,

Frishberg, Yaacov,

Garg, Pushkal P,

Simkova, Eva

2021

Overview

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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Publisher

Massachusetts Medical Society

Subject

Adolescent

/ Adult

/ Age

/ Calcinosis

/ Child

/ Creatinine

/ Creatinine - urine