Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

(2020) Daniel P. Judge et al.   CARDIOVASCULAR DRUGS AND THERAPY

Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis

(2019) Scott D. Solomon et al.   CIRCULATION

Highlights from Selected Cardiovascular Disease Prevention Studies Presented at the 2019 European Society of Cardiology Congress

(2019) Xiaoming Jia et al.   Current Atherosclerosis Reports

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

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

A new staging system for cardiac transthyretin amyloidosis

(2017) Julian D Gillmore et al.   EUROPEAN HEART JOURNAL

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

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

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Current and future treatment of amyloid diseases

(2016) M. Ankarcrona et al.   JOURNAL OF INTERNAL MEDICINE

Natural History of Wild-Type Transthyretin Cardiac Amyloidosis and Risk Stratification Using a Novel Staging System

(2016) Martha Grogan et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

Hereditary ATTR amyloidosis: a single-institution experience with 266 patients

(2015) Paul L. Swiecicki et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Evolving landscape in the management of transthyretin amyloidosis

(2015) Philip N. Hawkins et al.   ANNALS OF MEDICINE

Heart Failure Due to Age-Related Cardiac Amyloid Disease Associated With Wild-Type Transthyretin: A Prospective, Observational Cohort Study

(2015) Lawreen H. Connors et al.   CIRCULATION

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Cardiac amyloidosis: Updates in diagnosis and management

(2013) Dania Mohty et al.   Archives of Cardiovascular Diseases

Senile Systemic Amyloidosis: Clinical Features at Presentation and Outcome

(2013) Jennifer H. Pinney et al.   Journal of the American Heart Association

Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: The Transthyretin Amyloidosis Cardiac Study (TRACS)

(2012) Frederick L. Ruberg et al.   AMERICAN HEART JOURNAL

THAOS – The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis

(2012) Teresa Coelho et al.   CURRENT MEDICAL RESEARCH AND OPINION

Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective

(2012) C. Rapezzi et al.   EUROPEAN HEART JOURNAL

Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)

(2010) Gösta Holmgren et al.   CLINICAL GENETICS

Stem Cells and Liver Regeneration

(2009) Andrew W. Duncan et al.   GASTROENTEROLOGY