Sp771history of Renal and Ocular Findings in Primary Hyperoxaluria: From Oxalis Acetosella to the Mulberry Calculus, Ckd, Retinopathy and Systemic Oxalosis

Viggiano, Davide; Simonelli, Francesca; Capasso, Giovambattista; Santo, Natale De

doi:10.1093/ndt/gfy104.sp771

Nephrology Dialysis Transplantation

2018

DOI: 10.1093/ndt/gfy104.sp771

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Sp771history of Renal and Ocular Findings in Primary Hyperoxaluria: From Oxalis Acetosella to the Mulberry Calculus, Ckd, Retinopathy and Systemic Oxalosis

Davide Viggiano

Francesca Simonelli

Giovambattista Capasso

et al.

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“…It is estimated that the prevalence of PH is approximately 1–3 cases per 100,000 individuals in Europe − and also approximately 1–2 per 100,000 in the United States . There has been lack of treatment options for PH for a long time since the first case was diagnosed in 1925 . In additional to symptomatic relief, dual liver–kidney transplant is only option for curing PH, but it is an invasive and costly option.…”

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Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies

Liu

Zhao

Shah

et al. 2022

ACS Pharmacol. Transl. Sci.

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Due to the lack of treatment options for the genetic disease primary hyperoxaluria (PH), including three subtypes PH1, PH2, and PH3, caused by accumulation of oxalate forming kidney stones, there is an urgent need for the development of a drug therapy aside from siRNA drug lumasiran for patients with PH1. After the recent success of drug therapies based on small interfering RNA (siRNA), nedosiran is currently being developed for the treatment of three types of PH as a siRNA-based modality. Through specific inhibition of lactate dehydrogenase enzyme, the key enzyme in biosynthesis of oxalate in liver, phase 1, 2, and 3 clinical trials of nedosiran have achieved the desired primary end point of reduction of urinary oxalate levels in patients with PH1. More PH2 and PH3 patients need to be tested for efficacy. It has also produced a favorable secondary end point on safety and toxicity in PH patients. In addition to common injection site reactions that resolved spontaneously, no severe nedosiran treatment-associated adverse events were reported. Based on the positive results in the clinical studies, nedosiran is a candidate siRNA drug to treat PH patients.

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mentioning

confidence: 99%

Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies

Liu

Zhao

Shah

et al. 2022

ACS Pharmacol. Transl. Sci.

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Sp771history of Renal and Ocular Findings in Primary Hyperoxaluria: From Oxalis Acetosella to the Mulberry Calculus, Ckd, Retinopathy and Systemic Oxalosis

Cited by 1 publication

References 0 publications

Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies

Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies

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