Concepedia

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

Prajakta Badri, Xuemin Jiang, Anna Borodovsky, Nader Najafian, Jae Kim, Valerie A. Clausen, Varun Goel, Bahru Habtemariam, Gabriel J. Robbie

Clinical Pharmacokinetics · 2020 · 73 citations · 18 references

DOIFull text

Open access

Abstract

NCT02352493.

References

18

The Complement Inhibitor Eculizumab in Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen, Neal S. Young, Jörg Schubert et al. · New England Journal of Medicine · 2006 · 1.2K citations · Full text

Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria

Russell P. Rother, Scott A. Rollins, Christopher F. Mojcik et al. · Nature Biotechnology · 2007 · 752 citations

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Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria

Robert A. Brodsky, Neal S. Young, Elisabetta Antonioli et al. · Blood · 2007 · 640 citations

Effect of Eculizumab on Hemolysis and Transfusion Requirements in Patients with Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen, Claire Hall, Judith Marsh et al. · New England Journal of Medicine · 2004 · 631 citations · Full text

Genetic Variants in C5 and Poor Response to Eculizumab

Jun‐ichi Nishimura, Masaki Yamamoto, Shin Hayashi et al. · New England Journal of Medicine · 2014 · 392 citations · Full text