(Q34428314)

2 August 2017

An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis (English)

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2 August 2017

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ChEBI release 2020-05-28

Laura Obici

1

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Giampaolo Merlini

object named as

Giampaolo Merlini

series ordinal

2

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language of work or name

English

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publication date

8 July 2014

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Expert Opinion on Investigational Drugs

2 August 2017

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2 August 2017

23

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2 August 2017

9

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2 August 2017

1239-1251

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Self-propagation of pathogenic protein aggregates in neurodegenerative diseases

2 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Amyloid fibril protein nomenclature: 2012 recommendations from the Nomenclature Committee of the International Society of Amyloidosis

21 January 2018

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https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Familial amyloid polyneuropathy

21 January 2018

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https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

The Transthyretin Amyloidosis Outcomes Survey (THAOS) registry: design and methodology

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Sequence-dependent denaturation energetics: A major determinant in amyloid disease diversity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Liver transplantation and transthyretin amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Transthyretin internalization by sensory neurons is megalin mediated and necessary for its neuritogenic activity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Transthyretin protects Alzheimer's mice from the behavioral and biochemical effects of Abeta toxicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Transthyretin suppresses the toxicity of oligomers formed by misfolded proteins in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Mechanisms of transthyretin inhibition of β-amyloid aggregation in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Structural insight into pH-induced conformational changes within the native human transthyretin tetramer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Pathogenesis of and therapeutic strategies to ameliorate the transthyretin amyloidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

The biological and chemical basis for tissue-selective amyloid disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Transthyretin deposition in familial amyloidotic polyneuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Proteolytic cleavage of Ser52Pro variant transthyretin triggers its amyloid fibrillogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Sulfated glycosaminoglycans accelerate transthyretin amyloidogenesis by quaternary structural conversion

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Heparan sulfate/heparin promotes transthyretin fibrillization through selective binding to a basic motif in the protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Deposition of transthyretin in early stages of familial amyloidotic polyneuropathy: evidence for toxicity of nonfibrillar aggregates

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Tissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Only amyloidogenic intermediates of transthyretin induce apoptosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Inhibition of TTR aggregation-induced cell death--a new role for serum amyloid P component

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Transthyretin and familial amyloidotic polyneuropathy. Recent progress in understanding the molecular mechanism of neurodegeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Amyloidosis: pathogenesis and new therapeutic options.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Selective silencing of a mutant transthyretin allele by small interfering RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Safety and efficacy of RNAi therapy for transthyretin amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Modulation of lipoprotein metabolism by antisense technology: preclinical drug discovery methodology

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Prevention of transthyretin amyloid disease by changing protein misfolding energetics.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Synthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acid

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: a focus on the transthyretin amyloidoses.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Evaluating the binding selectivity of transthyretin amyloid fibril inhibitors in blood plasma

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Inhibition of human transthyretin aggregation by non-steroidal anti-inflammatory compounds: a structural and thermodynamic analysis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Iodine Atoms: A New Molecular Feature for the Design of Potent Transthyretin Fibrillogenesis Inhibitors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Small transthyretin (TTR) ligands as possible therapeutic agents in TTR amyloidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Human transthyretin in complex with iododiflunisal: structural features associated with a potent amyloid inhibitor

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

A Substructure Combination Strategy To Create Potent and Selective Transthyretin Kinetic Stabilizers That Prevent Amyloidogenesis and Cytotoxicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Biochemical and structural evaluation of highly selective 2-arylbenzoxazole-based transthyretin amyloidogenesis inhibitors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Potent Kinetic Stabilizers That Prevent Transthyretin-Mediated Cardiomyocyte Proteotoxicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Molecular dissection of Alzheimer's disease neuropathology by depletion of serum amyloid P component

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Amyloidogenesis in its biological environment: challenging a fundamental issue in protein misfolding diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Clearance of extracellular misfolded proteins in systemic amyloidosis: experience with transthyretin

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Interaction of the anthracycline 4'-iodo-4'-deoxydoxorubicin with amyloid fibrils: inhibition of amyloidogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

4 ′‐iodo‐4′‐Deoxydoxorubicin and tetracyclines disrupt transthyretin amyloid fibrils in vitro producing noncytotoxic species: screening for TTR fibril disrupters

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

4'-Iodo-4'-deoxydoxorubicin disrupts the fibrillar structure of transthyretin amyloid

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

A new face for old antibiotics: tetracyclines in treatment of amyloidoses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Doxycycline disrupts transthyretin amyloid: evidence from studies in a FAP transgenic mice model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Anti-apoptotic treatment reduces transthyretin deposition in a transgenic mouse model of Familial Amyloidotic Polyneuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Synergy of combined doxycycline/TUDCA treatment in lowering Transthyretin deposition and associated biomarkers: studies in FAP mouse models

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Effect of tetracyclines on the dynamics of formation and destructuration of beta2-microglobulin amyloid fibrils

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

C. elegans expressing human β2-microglobulin: a novel model for studying the relationship between the molecular assembly and the toxic phenotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Benefit of doxycycline treatment on articular disability caused by dialysis related amyloidosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Doxycycline reduces fibril formation in a transgenic mouse model of AL amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

EGCG remodels mature alpha-synuclein and amyloid-beta fibrils and reduces cellular toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

EGCG redirects amyloidogenic polypeptides into unstructured, off-pathway oligomers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

The main green tea polyphenol epigallocatechin-3-gallate counteracts semen-mediated enhancement of HIV infection

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Structural properties of EGCG-induced, nontoxic Alzheimer's disease Aβ oligomers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Insights into antiamyloidogenic properties of the green tea extract (-)-epigallocatechin-3-gallate toward metal-associated amyloid-β species

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Toward the molecular mechanism(s) by which EGCG treatment remodels mature amyloid fibrils

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Binding of epigallocatechin-3-gallate to transthyretin modulates its amyloidogenicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

The crystal structure of the green tea polyphenol (-)-epigallocatechin gallate-transthyretin complex reveals a novel binding site distinct from the thyroxine binding site

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Natural polyphenols inhibit different steps of the process of transthyretin (TTR) amyloid fibril formation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Epigallocatechin-3-gallate as a potential therapeutic drug for TTR-related amyloidosis: "in vivo" evidence from FAP mice models

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Green tea halts progression of cardiac transthyretin amyloidosis: an observational report

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Epigallocatechin-3-gallate (EGCG) for clinical trials: more pitfalls than promises?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Chitosan nanoparticles enhance the intestinal absorption of the green tea catechins (+)-catechin and (-)-epigallocatechin gallate

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Hepatotoxicity from green tea: a review of the literature and two unpublished cases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Binding and stabilization of transthyretin by curcumin.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Lysine-Specific Molecular Tweezers Are Broad-Spectrum Inhibitors of Assembly and Toxicity of Amyloid Proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Protection of primary neurons and mouse brain from Alzheimer's pathology by molecular tweezers

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

A novel "molecular tweezer" inhibitor of α-synuclein neurotoxicity in vitro and in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Molecular Tweezers Targeting Transthyretin Amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Sustained pharmacological depletion of serum amyloid P component in patients with systemic amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Antibodies to human serum amyloid P component eliminate visceral amyloid deposits

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Guideline of transthyretin-related hereditary amyloidosis for clinicians.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Tetramer Dissociation and Monomer Partial Unfolding Precedes Protofibril Formation in Amyloidogenic Transthyretin Variants

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F13543784.2014.922541

10.1517/13543784.2014.922541

21 January 2018

Identifiers

DOI

1 reference

2 August 2017

1 reference

PubMed ID

25003808