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

Pharmacokinetic and pharmacodynamic effects of ravulizumab and eculizumab on complement component 5 in adults with paroxysmal nocturnal haemoglobinuria: results of two phase 3 randomised, multicentre studies

(2020) Régis Peffault de Latour et al.   BRITISH JOURNAL OF HAEMATOLOGY

Pharmacology, Pharmacokinetics and Pharmacodynamics of Eculizumab, and Possibilities for an Individualized Approach to Eculizumab

(2019) Kioa Lente Wijnsma et al.   CLINICAL PHARMACOKINETICS

Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis

(2019) Linda L. Kusner et al.   Molecular Therapy-Methods & Clinical Development

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

(2018) Jennifer L.S. Willoughby et al.   MOLECULAR THERAPY

Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

(2018) Donald J. Foster et al.   MOLECULAR THERAPY

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Anticomplement C5 therapy with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome

(2015) Edwin K.S. Wong et al.   Translational Research

The global aHUS registry: methodology and initial patient characteristics

(2015) Christoph Licht et al.   BMC Nephrology

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

Genetic Variants in C5 and Poor Response to Eculizumab

(2014) Jun-ichi Nishimura et al.   NEW ENGLAND JOURNAL OF MEDICINE

Overview of Complement Activation and Regulation

(2013) Marina Noris et al.   SEMINARS IN NEPHROLOGY

Stem Cells and Liver Regeneration

(2009) Andrew W. Duncan et al.   GASTROENTEROLOGY