Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

(2020) Teresa Coelho et al.   Orphanet Journal of Rare Diseases

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

(2019) Scott D. Solomon et al.   CIRCULATION

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

(2019) David Adams et al.   Nature Reviews Neurology

Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

(2019) Alejandra González-Duarte et al.   JOURNAL OF NEUROLOGY

Frequency of and Prognostic Significance of Cardiac Involvement at Presentation in Hereditary Transthyretin-Derived Amyloidosis and the Value of N-Terminal Pro-B-Type Natriuretic Peptide

(2018) Sebastiaan H.C. Klaassen et al.   AMERICAN JOURNAL OF CARDIOLOGY

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

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

(2018) Mathew S. Maurer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis – Report from the Transthyretin Amyloidosis Outcome Survey (THAOS)

(2017) Arnt V. Kristen et al.   PLoS One

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

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

Prevalence and clinical phenotype of hereditary transthyretin amyloid cardiomyopathy in patients with increased left ventricular wall thickness

(2015) Thibaud Damy et al.   EUROPEAN HEART JOURNAL

Rapid progression of familial amyloidotic polyneuropathy

(2015) David Adams et al.   NEUROLOGY

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

(2015) Ole B Suhr et al.   Orphanet Journal of Rare Diseases

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

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

Genotype, echocardiography, and survival in familial transthyretin amyloidosis

(2013) Adelaide M. Arruda-Olson et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Cardiac amyloidosis: Updates in diagnosis and management

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

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Guideline of transthyretin-related hereditary amyloidosis for clinicians

(2013) Yukio Ando et al.   Orphanet Journal of Rare Diseases

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

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