Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

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

(2020) Bahru A. Habtemariam et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

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

(2019) Scott D. Solomon et al.   CIRCULATION

Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis

(2019) Xiaoping Zhang et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

(2019) David Adams et al.   Nature Reviews Neurology

Plasminogen activation triggers transthyretin amyloidogenesisin vitro

(2018) P. Patrizia Mangione et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

Unusual duplication mutation in a surface loop of human transthyretin leads to an aggressive drug-resistant amyloid disease

(2018) Elena S. Klimtchuk et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy

(2016) David Adams et al.   CURRENT OPINION IN NEUROLOGY

“Red-flag” symptom clusters in transthyretin familial amyloid polyneuropathy

(2016) Isabel Conceição et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

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

Rapid progression of familial amyloidotic polyneuropathy

(2015) David Adams et al.   NEUROLOGY

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

(2015) John L. Berk et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs

(2014) Adam Castaño et al.   HEART FAILURE REVIEWS

Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials

(2014) N. Suanprasert et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Cardiac amyloidosis: Updates in diagnosis and management

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

Precise assessment of noncompliance with the antihypertensive therapy in patients with resistant hypertension using toxicological serum analysis

(2013) Branislav Štrauch et al.   JOURNAL OF HYPERTENSION

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Amyloid Neuropathies

(2012) Susan C. Shin et al.   MOUNT SINAI JOURNAL OF MEDICINE

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

(2012) T. Coelho et al.   NEUROLOGY

Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant

(2011) P. T. Sattianayagam 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