Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial

BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period of the phase 3, multinational, open-label,...

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Detalles Bibliográficos
Autores principales: Hayes, Wesley, Sas, David J., Magen, Daniella, Shasha-Lavsky, Hadas, Michael, Mini, Sellier-Leclerc, Anne-Laure, Hogan, Julien, Ngo, Taylor, Sweetser, Marianne T., Gansner, John M., McGregor, Tracy L., Frishberg, Yaacov
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9925547/
https://www.ncbi.nlm.nih.gov/pubmed/35913563
http://dx.doi.org/10.1007/s00467-022-05684-1

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