Stiripentol fails to lower plasma oxalate in a dialysis-dependent PH1 patient

BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a multisystemic metabolic disorder caused by an excessive production of oxalate by the liver. The majority of patients presenting in early infancy have end-stage renal disease (ESRD). While awaiting the results of sRNAi trials, the current standard t...

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Detalles Bibliográficos
Autores principales: Kempf, Caroline, Pfau, Anja, Holle, Johannes, Müller-Schlüter, Karen, Bufler, Philip, Knauf, Felix, Müller, Dominik
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7385015/
https://www.ncbi.nlm.nih.gov/pubmed/32418144
http://dx.doi.org/10.1007/s00467-020-04585-5