CSF/plasma levels, transthyretin stabilisation and safety of multiple doses of tolcapone in subjects with hereditary ATTR amyloidosis

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

(2020) Francisca Pinheiro et al.   FEBS Journal

Blinded potency comparison of transthyretin kinetic stabilisers by subunit exchange in human plasma

(2020) Luke T. Nelson et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Interactions of tolcapone analogues as stabilizers of the amyloidogenic protein transthyretin

(2020) Valentina Loconte et al.   BIOORGANIC CHEMISTRY

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

A case of cerebral amyloid angiopathy-type hereditary ATTR amyloidosis with Y69H (p.Y89H) variant displaying transient focal neurological episodes as the main symptom

(2019) Yumi Yamada et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

First‐in‐Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers

(2019) Jonathan C. Fox et al.   Clinical Pharmacology in Drug Development

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

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

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

Cerebral amyloid angiopathy in posttransplant patients with hereditary ATTR amyloidosis

(2016) Yoshiki Sekijima et al.   NEUROLOGY

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

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

Neuropathologic analysis of Tyr69His TTR variant meningovascular amyloidosis with dementia

(2015) Jennifer L. Ziskin et al.   Acta Neuropathologica Communications

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

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

Quantification of Quaternary Structure Stability in Aggregation-Prone Proteins under Physiological Conditions: The Transthyretin Case

(2014) Lei Z. Robinson et al.   BIOCHEMISTRY

Transthyretin-type cerebral amyloid angiopathy: a serious complication in post-transplant patients with familial amyloid polyneuropathy

(2014) Y. Sekijima   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

CNS involvement in V30M transthyretin amyloidosis: clinical, neuropathological and biochemical findings

(2014) L. F. Maia et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

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

Report of five rare or previously unknown amyloidogenic transthyretin mutations disclosed in Sweden

(2010) Ole B. Suhr et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Oculoleptomeningeal amyloidosis in 3 individuals with the transthyretin variant Tyr69His

(2009) Kelly Schweitzer et al.   CANADIAN JOURNAL OF OPHTHALMOLOGY-JOURNAL CANADIEN D OPHTALMOLOGIE