Results 1 to 10 of about 4,438 (134)

LNP-mediated in vivo base editing corrects Agxt to cure primary hyperoxaluria type 1. [PDF]

Clin Transl Med
Zhang D, Zheng R, Chen Z, Chen X, Yang L, Huo Y, Zhao Y, Huang J, Zhang D, Yin S, Li D, Geng H.   +11 more
europepmc   +1 more source

Isolated Kidney Transplant in Primary Hyperoxaluria-1 Enabled by Small Interfering RNA (siRNA) Therapy. Is It Time for Change? Case Report and Review of the Literature. [PDF]

Pediatr Transplant
Habeeb SM, Simkova E, Al-Akash S, Awad HS, Yamin H, Eid LA, Aldhaheri WS, Concepcion W, Bitzan M.   +8 more
europepmc   +1 more source

Surgical Management of Pediatric Primary Hyperoxaluria Type 1: An Eight-Patient Case Series in the Pre-siRNA Era. [PDF]

Pediatr Transplant
Finer G, Darani AN, Switalski MG, Ward SE, Melin-Aldana H, Superina R.   +5 more
europepmc   +1 more source

Natural History of Advanced Primary Hyperoxaluria Type 1: A Retrospective Study. [PDF]

Kidney Med
Lieske JC, Groothoff JW, Frishberg Y, Garrelfs SF, Milliner DS, Magen D, Sellier-Leclerc AL, Shasha-Lavsky H, Bakkaloǧlu S, Lemoine S, Fuster DG, Devresse A, Cytter-Kuint R, Kajbaf F, Du W, Gansner JM.   +15 more
europepmc   +1 more source

Urine Oxalate Excretion and CKD Stage in Patients With Primary Hyperoxaluria Type 1. [PDF]

Am J Kidney Dis
Vaughan LE, Schulte PJ, Knauf F, Sas DJ, Milliner DS, Lieske JC.   +5 more
europepmc   +1 more source

Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1. [PDF]

Clin J Am Soc Nephrol
Frishberg Y, Saland JM, Lieske JC, Du W, Coenen M, Hogan J, Sellier-Leclerc AL, Groothoff JW, Kaspar C, Gansner JM, Hulton SA, ILLUMINATE-A study investigators.   +11 more
europepmc   +1 more source

Genetic Correction of the Most Common Mutation Causing Primary Hyperoxaluria Restores Enzyme Localization and Oxalate Metabolism. [PDF]

J Inherit Metab Dis
Keskinen T, Jalil S, Gümüşoğlu I, Juutila J, Kestilä N, Kuuluvainen E, Hietakangas V, Balboa D, Wartiovaara K, Hyvönen ME.   +9 more
europepmc   +1 more source

The Dawn of Precision Medicine in Pediatric Nephrology: Lumasiran and the Era of siRNA Therapies for Primary Hyperoxaluria Type 1. [PDF]

J Pers Med
Dotis J, Fourikou M.
europepmc   +1 more source

Real-world burden of primary hyperoxaluria with chronic kidney disease in the United States: a retrospective administrative claims analysis. [PDF]

BMC Nephrol
Goldfarb DS, Modersitzki F, Skaar JR, Li O, Salem S, Chen JV.   +5 more
europepmc   +1 more source

Primary hyperoxaluria: insights into its clinical presentation, genetic mutations, and transplantation outcomes in a pediatric population in a tertiary care center. [PDF]

Orphanet J Rare Dis
Sayed B, Alhuthil R, Saadeh S, Al-Shareef T, Alhassoun I, Al-Sabban E.   +5 more
europepmc   +1 more source