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1958 1
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1985 2
1987 2
1988 2
1989 2
1990 2
1991 1
1992 2
1993 4
1994 3
1995 5
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1998 4
1999 6
2000 1
2001 4
2002 5
2003 2
2004 4
2005 5
2006 7
2007 1
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2009 4
2010 3
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2012 3
2013 5
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2015 10
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2023 12
2024 2

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191 results

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Page 1
Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial.
Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Michael M, et al. Am J Kidney Dis. 2023 Feb;81(2):145-155.e1. doi: 10.1053/j.ajkd.2022.05.012. Epub 2022 Jul 14. Am J Kidney Dis. 2023. PMID: 35843439 Free article. Clinical Trial.
RATIONALE & OBJECTIVE: Lumasiran reduces urinary and plasma oxalate (POx) in patients with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function. ...TRIAL REGISTRATION: Registered at ClinicalTrials.gov with study number NCT04152200 and at E …
RATIONALE & OBJECTIVE: Lumasiran reduces urinary and plasma oxalate (POx) in patients with primary hyperoxaluria type 1 (P …
Phenotype-Genotype Correlations and Estimated Carrier Frequencies of Primary Hyperoxaluria.
Hopp K, Cogal AG, Bergstralh EJ, Seide BM, Olson JB, Meek AM, Lieske JC, Milliner DS, Harris PC; Rare Kidney Stone Consortium. Hopp K, et al. J Am Soc Nephrol. 2015 Oct;26(10):2559-70. doi: 10.1681/ASN.2014070698. Epub 2015 Feb 2. J Am Soc Nephrol. 2015. PMID: 25644115 Free PMC article.
Primary hyperoxaluria (PH) is a rare autosomal recessive disease characterized by oxalate accumulation in the kidneys and other organs. ...We estimated PH to be approximately three times less prevalent among African Americans than among European Americans because of …
Primary hyperoxaluria (PH) is a rare autosomal recessive disease characterized by oxalate accumulation in the kidneys and othe …
Primary hyperoxaluria.
Lorenzo V, Torres A, Salido E. Lorenzo V, et al. Nefrologia. 2014 May 21;34(3):398-412. doi: 10.3265/Nefrologia.pre2014.Jan.12335. Epub 2014 Apr 30. Nefrologia. 2014. PMID: 24798559 Free article. Review. English, Spanish.
Primary hyperoxaluria (PH) occurs due to an autosomal recessive hereditary disorder of the metabolism of glyoxylate, which causes excessive oxalate production. ...Treatment by dialysis is ineffective in treating excess oxalate. After the kidney transplant, we normal …
Primary hyperoxaluria (PH) occurs due to an autosomal recessive hereditary disorder of the metabolism of glyoxylate, which cau …
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
Conjugation of oligonucleotide therapeutics, including small interfering RNAs (siRNAs) or antisense oligonucleotides, to N-acetylgalactosamine (GalNAc) ligands has become the primary strategy for hepatocyte-targeted delivery, and with the recent approvals of GIVLAARI (givosiran) …
Conjugation of oligonucleotide therapeutics, including small interfering RNAs (siRNAs) or antisense oligonucleotides, to N-acetylgalactosami …
Primary hyperoxaluria type 1 in children: clinical and laboratory manifestations and outcome.
Wannous H. Wannous H. Pediatr Nephrol. 2023 Aug;38(8):2643-2648. doi: 10.1007/s00467-023-05917-x. Epub 2023 Mar 14. Pediatr Nephrol. 2023. PMID: 36917293
BACKGROUND: Primary hyperoxaluria (PH) results from genetic mutations in different genes of glyoxylate metabolism, which cause significant increases in production of oxalate by the liver. This study aimed to report clinical and laboratory manifestations and outcome …
BACKGROUND: Primary hyperoxaluria (PH) results from genetic mutations in different genes of glyoxylate metabolism, which cause …
Urinary proteome in inherited nephrolithiasis.
Capolongo G, Zacchia M, Perna A, Viggiano D, Capasso G. Capolongo G, et al. Urolithiasis. 2019 Feb;47(1):91-98. doi: 10.1007/s00240-018-01104-y. Epub 2018 Dec 18. Urolithiasis. 2019. PMID: 30564846 Review.
In the last decades, proteomics has been largely applied to the Nephrology field, with the double aim to (1) elucidate the biological processes underlying renal diseases; (2) identify disease-specific biomarkers, predictor factors of therapeutic efficacy and prognostic fac …
In the last decades, proteomics has been largely applied to the Nephrology field, with the double aim to (1) elucidate the biological proces …
Clinical and economic impact of primary hyperoxaluria: a retrospective claims analysis.
Mucha L, Hoppe B, Silber A, Wang Z, Miyasato G, Skaar JR, Langman C. Mucha L, et al. J Manag Care Spec Pharm. 2022 Mar;28(3):316-323. doi: 10.18553/jmcp.2022.28.3.316. J Manag Care Spec Pharm. 2022. PMID: 35199581 Free PMC article.
BACKGROUND: Primary hyperoxaluria (PH) is a family of rare, life-threatening genetic liver disorders characterized by elevated production and excretion of oxalate. ...PH patients had a significantly higher mean Charlson Comorbidity Index composite score than …
BACKGROUND: Primary hyperoxaluria (PH) is a family of rare, life-threatening genetic liver disorders characterized by elevated …
End Points for Clinical Trials in Primary Hyperoxaluria.
Milliner DS, McGregor TL, Thompson A, Dehmel B, Knight J, Rosskamp R, Blank M, Yang S, Fargue S, Rumsby G, Groothoff J, Allain M, West M, Hollander K, Lowther WT, Lieske JC. Milliner DS, et al. Clin J Am Soc Nephrol. 2020 Jul 1;15(7):1056-1065. doi: 10.2215/CJN.13821119. Epub 2020 Mar 12. Clin J Am Soc Nephrol. 2020. PMID: 32165440 Free PMC article.
Patients with primary hyperoxaluria experience kidney stones from a young age and can develop progressive oxalate nephropathy. ...Primary hyperoxaluria registry data suggest that eGFR decline in most patients is gradual, but can be unpredictable. ...
Patients with primary hyperoxaluria experience kidney stones from a young age and can develop progressive oxalate nephropathy. …
Natural history of urine and plasma oxalate in children with primary hyperoxaluria type 1.
Sas DJ, Mara K, Mehta RA, Seide BM, Banks CJ, Danese DS, McGregor TL, Lieske JC, Milliner DS. Sas DJ, et al. Pediatr Nephrol. 2024 Jan;39(1):141-148. doi: 10.1007/s00467-023-06074-x. Epub 2023 Jul 17. Pediatr Nephrol. 2024. PMID: 37458799 Free PMC article.
BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a rare, severe genetic disease causing increased hepatic oxalate production resulting in urinary stone disease, nephrocalcinosis, and often progressive chronic kidney disease. Little is known about the natural histor …
BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a rare, severe genetic disease causing increased hepatic oxalate production …
Primary hyperoxaluria type 1: practical and ethical issues.
Cochat P, Groothoff J. Cochat P, et al. Pediatr Nephrol. 2013 Dec;28(12):2273-81. doi: 10.1007/s00467-013-2444-5. Epub 2013 Mar 14. Pediatr Nephrol. 2013. PMID: 23494551 Review.
Primary hyperoxaluria type 1 (PH1) is a rare inborn error of glyoxylate metabolism of autosomal recessive inheritance, leading to progressive systemic oxalate storage (named 'oxalosis') with a high rate of morbidity and mortality, as well as an unacceptable quality …
Primary hyperoxaluria type 1 (PH1) is a rare inborn error of glyoxylate metabolism of autosomal recessive inheritance, leading …
191 results