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

INOTERSEN IMPROVES QUALITY OF LIFE IN PATIENTS WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPATHY AND CARDIOMYOPATHY: RESULTS OF THE PHASE 3 STUDY NEURO-TTR

(2018) Mathew S. Maurer et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Drug-induced liver injury: recent advances in diagnosis and risk assessment

(2017) Gerd A Kullak-Ublick et al.   GUT

Parkinson’s Disease: From Pathogenesis to Pharmacogenomics

(2017) Ramón Cacabelos   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Inhibition of the mechano-enzymatic amyloidogenesis of transthyretin: role of ligand affinity, binding cooperativity and occupancy of the inner channel

(2017) Guglielmo Verona et al.   Scientific Reports

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

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

Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe

(2016) Yesim Parman et al.   CURRENT OPINION IN NEUROLOGY

Current and Future Treatment Approaches in Transthyretin Familial Amyloid Polyneuropathy

(2016) Philippe Kerschen et al.   Current Treatment Options in Neurology

TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies

(2016) David Adams et al.   EXPERT OPINION ON PHARMACOTHERAPY

Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study

(2016) Violaine Planté-Bordeneuve et al.   JOURNAL OF NEUROLOGY

Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

(2016) Ricardo Sant'Anna et al.   Nature Communications

Liver transplantation in transthyretin amyloidosis: Issues and challenges

(2015) Andreia Carvalho et al.   LIVER TRANSPLANTATION

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

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

Update in the diagnosis and management of transthyretin familial amyloid polyneuropathy

(2014) Violaine Plante-Bordeneuve   JOURNAL OF NEUROLOGY

Incat disability score: A critical analysis of its measurement properties

(2014) Ari Breiner et al.   MUSCLE & NERVE

Recent advances in transthyretin amyloidosis therapy

(2014) Mitsuharu Ueda et al.   Translational Neurodegeneration

Changes in pathological and biochemical findings of systemic tissue sites in familial amyloid polyneuropathy more than 10 years after liver transplantation

(2013) T. Oshima et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

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

(2012) T. Coelho et al.   NEUROLOGY

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade

(2012) C. E. Bulawa et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Familial amyloid polyneuropathy

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

Meta-analysis of the comparative efficacy and safety of adjuvant treatment to levodopa in later Parkinson's disease

(2011) Rebecca Stowe et al.   MOVEMENT DISORDERS

Progression of transthyretin amyloid neuropathy after liver transplantation

(2010) J. J. Liepnieks et al.   NEUROLOGY