{"id":"21d1dcf90390","type":"article","url":"https://hartvaat.nl/2026/01/22/primaire-hyperoxalurie-van-trials-naar-real-world-data-en-pijplijntherapieen/","title":"Primaire hyperoxalurie: van trials naar real-world data en pijplijntherapieën","title_en":"Primary hyperoxaluria(s): from trials to real-life data and pipeline therapies","category":"chronische nierziekte","category_label":"Nierziekte","professions":["internist"],"tags":[],"journal":"Kidney International","doi":"https://www.kidney-international.org/article/S0085-2538(26)00053-0/fulltext","source_url":"https://doi.org/https://www.kidney-international.org/article/S0085-2538(26)00053-0/fulltext","authors":["Justine Bacchetta","Cécile Acquaviva-Bourdain","Nadia Abid","Anne Laure Sellier Leclerc","Arnaud Molin","Sandrine Lemoine"],"significance":5,"published":"2026-01-22","source_date":"2026-01-22","image":"","kennis":[],"congress":"","summary_en":"This review summarises the evolving landscape of primary hyperoxaluria treatment, from clinical trials to real-world data and pipeline therapies. siRNA-based hepatic enzyme silencing has revolutionised management of these rare disorders of glyoxylate metabolism.","created":"2026-07-03T10:25:30Z","updated":"2026-07-03T13:24:55Z","licence":"Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.","body_markdown":"Primaire hyperoxalurieën zijn zeldzame autosomaal recessieve stoornissen van het glyoxalatmetabolisme die leiden tot overmatige oxalaatproductie, nierstenen en nierfalen. Dit overzicht bespreekt de laatste trials, real-world gegevens en therapieën in ontwikkeling.","abstract_original":"Primary hyperoxalurias (PHs) are a group of rare autosomal recessive disorders of glyoxylate metabolism leading to excessive oxalate production, recurrent nephrolithiasis, nephrocalcinosis, and progression to kidney failure with systemic oxalosis in the most severe forms. Until recently, treatment options were limited to conservative measures and double liver/kidney transplantation. The advent of small interfering RNA therapies has revolutionized the field by enabling targeted hepatic enzyme silencing via GalNAc-conjugated delivery involved in oxalate synthesis."}