Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis

Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis

(2021) Jan M. Griffin et al.   CIRCULATION RESEARCH

Expert consensus on the monitoring of transthyretin amyloid cardiomyopathy

(2021) Pablo Garcia‐Pavia et al.   EUROPEAN JOURNAL OF HEART FAILURE

A machine learning model for identifying patients at risk for wild-type transthyretin amyloid cardiomyopathy

(2021) Ahsan Huda et al.   Nature Communications

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

A human antibody selective for transthyretin amyloid removes cardiac amyloid through phagocytic immune cells

(2021) Aubin Michalon et al.   Nature Communications

Spironolactone in Patients With an Echocardiographic HFpEF Phenotype Suggestive of Cardiac Amyloidosis

(2021) Brett W. Sperry et al.   JACC-Heart Failure

Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression

(2021) David Adams et al.   Orphanet Journal of Rare Diseases

ATTR Amyloidosis: Current and Emerging Management Strategies

(2021) Jan M. Griffin et al.   JACC: CardioOncology

Tolcapone, a potent aggregation inhibitor for the treatment of familial leptomeningeal amyloidosis

(2020) Francisca Pinheiro et al.   FEBS Journal

Diagnosis and treatment of transthyretin‐related amyloidosis cardiomyopathy

(2020) Catherine Teng et al.   CLINICAL CARDIOLOGY

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

Gateway and journey of patients with cardiac amyloidosis

(2020) Daniel Dang et al.   ESC Heart Failure

Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial ( ATTR‐ACT ) and long‐term extension study

(2020) Thibaud Damy et al.   EUROPEAN JOURNAL OF HEART FAILURE

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

(2020) David Adams et al.   LANCET NEUROLOGY

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

(2019) Scott D. Solomon et al.   CIRCULATION

Transthyretin stabilization activity of the catechol-O-methyltransferase inhibitor tolcapone (SOM0226) in hereditary ATTR amyloidosis patients and asymptomatic carriers: proof-of-concept study

(2019) Josep Gamez et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

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

(2019) David Adams et al.   Nature Reviews Neurology

Diagnosis and treatment of gastrointestinal dysfunction in hereditary TTR amyloidosis

(2019) Laura Obici et al.   CLINICAL AUTONOMIC RESEARCH

Therapeutic Approaches for Peripheral and Central Neuropathic Pain

(2019) Délia Szok et al.   BEHAVIOURAL NEUROLOGY

Cerebrospinal fluid and vitreous body exposure to orally administered tafamidis in hereditary ATTRV30M (p.TTRV50M) amyloidosis patients

(2018) Cecilia Monteiro et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Outrageous prices of orphan drugs: a call for collaboration

(2018) Lucio Luzzatto et al.   LANCET

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

Amyloid seeding of transthyretin by ex vivo cardiac fibrils and its inhibition

(2018) Lorena Saelices et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

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

6MWT performance correlates with peripheral neuropathy but not with cardiac involvement in patients with hereditary transthyretin amyloidosis (hATTR)

(2018) Gian Luca Vita et al.   NEUROMUSCULAR DISORDERS

Novel Antibody for the Treatment of Transthyretin Amyloidosis

(2016) Akihiko Hosoi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Evolving landscape in the management of transthyretin amyloidosis

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

The Val30Met familial amyloid polyneuropathy specific Rasch-built overall disability scale (FAP-RODS©)

(2015) Mariëlle H. J. Pruppers et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Liver Transplantation for Hereditary Transthyretin Amyloidosis

(2015) Bo-Göran Ericzon et al.   TRANSPLANTATION

Amyloid fibrils containing fragmented ATTR may be the standard fibril composition in ATTR amyloidosis

(2013) Elisabet Ihse et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin

(2013) S. C. Penchala et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis

(2013) Giampaolo Merlini et al.   Journal of Cardiovascular Translational Research

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

(2012) T. Coelho et al.   NEUROLOGY

The diflunisal trial: update on study drug tolerance and disease progression

(2011) J. L. Berk et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Familial amyloid polyneuropathy

(2011) Violaine Planté-Bordeneuve et al.   LANCET NEUROLOGY