Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial

Introduction: Patients with primary hyperoxaluria type 1 (PH1), a genetic disorder associated with hepatic oxalate overproduction, frequently experience recurrent kidney stones and worsening kidney function. Lumasiran is indicated for the treatment of PH1 to lower urinary and plasma oxalate (POx). M...

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Hauptverfasser:	Jeffrey M. Saland, John C. Lieske, Jaap W. Groothoff, Yaacov Frishberg, Hadas Shasha-Lavsky, Daniella Magen, Shabbir H. Moochhala, Eva Simkova, Martin Coenen, Wesley Hayes, Julien Hogan, Anne-Laure Sellier-Leclerc, Richard Willey, John M. Gansner, Sally-Anne Hulton
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Elsevier 2024-07-01
Schriftenreihe:	Kidney International Reports
Schlagworte:	lumasiran oxalate primary hyperoxaluria type 1 rare disease renal RNA interference
Online-Zugang:	http://www.sciencedirect.com/science/article/pii/S2468024924016838
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