4.6 Editorial Material

Primary hyperoxaluria type 1: time for prime time?

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Pharmacology & Pharmacy

Lumasiran: First Approval

Lesley J. Scott et al.

Summary: Lumasiran is a drug developed for the treatment of primary hyperoxaluria type 1 by targeting a specific gene to inhibit the synthesis of toxic metabolites. It received approvals in the EU and USA, marking its global recognition.
Article Medicine, General & Internal

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Sander F. Garrelfs et al.

Summary: Primary hyperoxaluria type 1 is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested the efficacy of lumasiran, an RNAi therapeutic agent, in reducing hepatic oxalate production. The results showed that lumasiran significantly reduced urinary oxalate excretion, alleviating the cause of kidney failure in PH1 patients.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Urology & Nephrology

Patients with primary hyperoxaluria type 2 have significant morbidity and require careful follow-up

Sander F. Garrelfs et al.

KIDNEY INTERNATIONAL (2019)

Article Urology & Nephrology

Predictors of Incident ESRD among Patients with Primary Hyperoxaluria Presenting Prior to Kidney Failure

Fang Zhao et al.

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2016)

Review Medicine, General & Internal

Primary Hyperoxaluria

Pierre Cochat et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Article Transplantation

Primary hyperoxaluria Type 1: indications for screening and guidance for diagnosis and treatment

Pierre Cochat et al.

NEPHROLOGY DIALYSIS TRANSPLANTATION (2012)

Article Multidisciplinary Sciences

Alanine-glyoxylate aminotransferase-deficient mice, a model for primary hyperoxaluria that responds to adenoviral gene transfer

Eduardo C. Salido et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)