Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

BACKGROUND: Cemdisiran, an N-acetylgalactosamine (GalNAc) conjugated RNA interference (RNAi) therapeutic, is currently under development for the treatment of complement-mediated diseases by suppressing liver production of complement 5 (C5) protein. This study was designed to evaluate the safety, tol...

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Detalles Bibliográficos
Autores principales: Badri, Prajakta, Jiang, Xuemin, Borodovsky, Anna, Najafian, Nader, Kim, Jae, Clausen, Valerie A., Goel, Varun, Habtemariam, Bahru, Robbie, Gabriel J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2020
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9203406/
https://www.ncbi.nlm.nih.gov/pubmed/33047216
http://dx.doi.org/10.1007/s40262-020-00940-9

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