Nedosiran in primary hyperoxaluria subtype 3: results from a phase I, single-dose study (PHYOX4)

Nedosiran is an N-acetyl-D-galactosamine (GalNAc)–conjugated RNA interference agent targeting hepatic lactate dehydrogenase (encoded by the LDHA gene), the putative enzyme mediating the final step of oxalate production in all three genetic subtypes of primary hyperoxaluria (PH). This phase I study a...

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Detalles Bibliográficos
Autores principales: Goldfarb, David S., Lieske, John C., Groothoff, Jaap, Schalk, Gesa, Russell, Kerry, Yu, Shuli, Vrhnjak, Blaz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10147791/
https://www.ncbi.nlm.nih.gov/pubmed/37118061
http://dx.doi.org/10.1007/s00240-023-01453-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10147791/
https://www.ncbi.nlm.nih.gov/pubmed/37118061
http://dx.doi.org/10.1007/s00240-023-01453-3

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