Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1

(2021) Yaacov Frishberg et al.   Clinical Journal of the American Society of Nephrology

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

(2021) Sander F. Garrelfs et al.   NEW ENGLAND JOURNAL OF MEDICINE

Endpoints for Clinical Trials in Primary Hyperoxaluria

(2020) Dawn S. Milliner et al.   Clinical Journal of the American Society of Nephrology

Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria

(2020) Tracy L McGregor et al.   eLife

Recovery From Dialysis in Patients With Primary Hyperoxaluria Type 1 Treated With Pyridoxine: A Report of 3 Cases

(2020) Elizabeth C. Lorenz et al.   AMERICAN JOURNAL OF KIDNEY DISEASES

The current state and future directions of RNAi-based therapeutics

(2019) Ryan L. Setten et al.   NATURE REVIEWS DRUG DISCOVERY

RNAi therapeutic and its innovative biotechnological evolution

(2019) Yuhua Weng et al.   BIOTECHNOLOGY ADVANCES

Recent advances in the identification and management of inherited hyperoxalurias

(2018) David J. Sas et al.   Urolithiasis

Glycolate oxidase deficiency in a patient with congenital hyperinsulinism and unexplained hyperoxaluria

(2017) Oliver Clifford-Mobley et al.   PEDIATRIC NEPHROLOGY

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

(2015) F. Zhao et al.   Clinical Journal of the American Society of Nephrology

Phenotype-Genotype Correlations and Estimated Carrier Frequencies of Primary Hyperoxaluria

(2015) K. Hopp et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Nephrocalcinosis is a risk factor for kidney failure in primary hyperoxaluria

(2015) Xiaojing Tang et al.   KIDNEY INTERNATIONAL

Sustained Pyridoxine Response in Primary Hyperoxaluria Type 1 Recipients of Kidney Alone Transplant

(2014) E. C. Lorenz et al.   AMERICAN JOURNAL OF TRANSPLANTATION

Mutations inHAO1encoding glycolate oxidase cause isolated glycolic aciduria

(2014) Yaacov Frishberg et al.   JOURNAL OF MEDICAL GENETICS

Data from a large European study indicate that the outcome of primary hyperoxaluria type 1 correlates with the AGXT mutation type

(2014) Giorgia Mandrile et al.   KIDNEY INTERNATIONAL

Primary Hyperoxaluria

(2013) Pierre Cochat et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pre-emptive liver transplantation for primary hyperoxaluria (PH-I) arrests long-term renal function deterioration

(2010) M. T. P. Perera et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Excellent renal function and reversal of nephrocalcinosis 8 years after isolated liver transplantation in an infant with primary hyperoxaluria type 1

(2010) Mónica Galanti et al.   PEDIATRIC NEPHROLOGY

New Equations to Estimate GFR in Children with CKD

(2009) G. J. Schwartz et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Genotype–phenotype correlation in primary hyperoxaluria type 1: the p.Gly170Arg AGXT mutation is associated with a better outcome

(2009) Jérôme Harambat et al.   KIDNEY INTERNATIONAL

Twenty-four Hour and Spot Urine Metabolic Evaluations: Correlations Versus Agreements

(2009) Yet Hoi Hong et al.   UROLOGY

Mechanism-Based Concepts of Size and Maturity in Pharmacokinetics

(2007) B.J. Anderson et al.   Annual Review of Pharmacology and Toxicology