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Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria

Complement dysregulation underpins the physiopathology of paroxysmal nocturnal hemoglobinuria (PNH). Cemdisiran, an RNA interference investigational treatment, silences complement component 5 (C5) expression in the liver. Previously reported results showed sustained reduction in C5 levels following...

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Detalles Bibliográficos
Autores principales:	Gaya, Anna, Munir, Talha, Urbano‐Ispizua, Alvaro, Griffin, Morag, Taubel, Jorg, Bush, Jim, Bhan, Ishir, Borodovsky, Anna, Wang, Yue, Badri, Prajakta, Garg, Pushkal
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2023
Materias:	Sickle Cell, Thrombosis, and Classical Haematology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10435727/ https://www.ncbi.nlm.nih.gov/pubmed/37601837 http://dx.doi.org/10.1002/jha2.748

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