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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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Автори: 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
Формат: Artigo
Мова:Inglês
Опубліковано: Elsevier 2024-07-01
Серія:Kidney International Reports
Предмети:
lumasiran
oxalate
primary hyperoxaluria type 1
rare disease
renal
RNA interference
Онлайн доступ:http://www.sciencedirect.com/science/article/pii/S2468024924016838
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