Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis

MS Maurer, S Bokhari, T Damy, S Dorbala… - Circulation: Heart …, 2019 - Am Heart Assoc
Cardiomyopathy is a manifestation of transthyretin amyloidosis (ATTR), which is an
underrecognized systemic disease whereby the transthyretin protein misfolds to form fibrils …

Therapeutic RNA-silencing oligonucleotides in metabolic diseases

A Goga, M Stoffel - Nature Reviews Drug Discovery, 2022 - nature.com
Recent years have seen unprecedented activity in the development of RNA-silencing
oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and …

[HTML][HTML] Structural polymorphism of amyloid fibrils in ATTR amyloidosis revealed by cryo-electron microscopy

BA Nguyen, V Singh, S Afrin, A Yakubovska… - Nature …, 2024 - nature.com
ATTR amyloidosis is caused by the deposition of transthyretin in the form of amyloid fibrils in
virtually every organ of the body, including the heart. This systemic deposition leads to a …

Cardiac amyloidosis

MW Bloom, PD Gorevic - Annals of Internal Medicine, 2023 - acpjournals.org
Amyloidosis is a pathologic and clinical condition resulting from the accumulation of
insoluble aggregates of misfolded proteins in tissues. Extracellular deposition of amyloid …

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

L Saelices, K Chung, JH Lee, W Cohn… - Proceedings of the …, 2018 - National Acad Sciences
Each of the 30 human amyloid diseases is associated with the aggregation of a particular
precursor protein into amyloid fibrils. In transthyretin amyloidosis (ATTR), mutant or wild-type …

Biomolecular assemblies: moving from observation to predictive design

CJ Wilson, AS Bommarius, JA Champion… - Chemical …, 2018 - ACS Publications
Biomolecular assembly is a key driving force in nearly all life processes, providing structure,
information storage, and communication within cells and at the whole organism level. These …

Hip and knee arthroplasty are common among patients with transthyretin cardiac amyloidosis, occurring years before cardiac amyloid diagnosis: can we identify …

J Rubin, J Alvarez, S Teruya, A Castano, RA Lehman… - Amyloid, 2017 - Taylor & Francis
Transthyretin cardiac amyloidosis (ATTR-CA) causes a restrictive cardiomyopathy in older
adults, often diagnosed at advanced stages when emerging therapies in late phase clinical …

[HTML][HTML] Modulation of the mechanisms driving transthyretin amyloidosis

F Bezerra, MJ Saraiva, MR Almeida - Frontiers in Molecular …, 2020 - frontiersin.org
Transthyretin (TTR) amyloidoses are systemic diseases associated with TTR aggregation
and extracellular deposition in tissues as amyloid. The most frequent and severe forms of …

Myocardial contraction fraction by M-mode echocardiography is superior to ejection fraction in predicting mortality in transthyretin amyloidosis

J Rubin, DE Steidley, M Carlsson, ML Ong… - Journal of cardiac …, 2018 - Elsevier
Background Transthyretin amyloidosis (ATTR) is often associated with cardiac involvement
manifesting as conduction disease as well as restrictive cardiomyopathy causing heart …

Inotersen for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis

MA Gertz, M Scheinberg… - Expert review of …, 2019 - Taylor & Francis
Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is an
underdiagnosed, progressive, and fatal multisystemic disease with a heterogenous clinical …