Case Report: Sustained Efficacy of Lumasiran at 18 Months in Primary Hyperoxaluria Type 1

Background: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate, ultimately responsible for kidney stones, kidney failure and systemic oxalosis. Lumasiran, is a liver-directed RNA interference therapeutic agent. It has been shown to reduce hepatic...

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Detalles Bibliográficos
Autores principales: Chiodini, Benedetta, Tram, Nathalie, Adams, Brigitte, Hennaut, Elise, Lolin, Ksenija, Ismaili, Khalid
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Pediatrics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8767018/
https://www.ncbi.nlm.nih.gov/pubmed/35071135
http://dx.doi.org/10.3389/fped.2021.791616

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8767018/
https://www.ncbi.nlm.nih.gov/pubmed/35071135
http://dx.doi.org/10.3389/fped.2021.791616

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