Results 61 to 70 of about 21,545 (230)

Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study

ESC Heart Failure, Volume 12, Issue 2, Page 1203-1216, April 2025.
The 366 patients diagnosed with transthyretin amyloidosis cardiomyopathy (ATTR‐CM) were analyzed regarding their clinical characteristics in the first year after approval of tafamidis 61 mg for ATTR‐CM in Germany. Nearly two‐thirds of the patients were in an advanced disease stage and 64% met the key criteria of the “Transthyretin Amyloidosis ...
Richard J. Nies, Svenja Ney, Ingrid Kindermann, Yvonne Bewarder, Angela Zimmer, Fabian Knebel, Katrin Hahn, Sebastian Spethmann, Peter Luedike, Lars Michel, Tienush Rassaf, Maria Papathanasiou, Stefan Störk, Vladimir Cejka, Amin Polzin, Fabian Voss, Malte Kelm, Bernhard Unsöld, Christine Meindl, Michael Paulus, Ali Yilmaz, Bishwas Chamling, Caroline Morbach, Roman Pfister   +23 more
wiley   +1 more source

The impact of limited healthcare access among patients with light chain and transthyretin amyloidosis: real-world survey during COVID-19 lockdown period in France

Orphanet Journal of Rare Diseases
Background The containment strategies during the COVID-19 pandemic between December 2019 and 2022 significantly disrupted the healthcare system. Cardiac amyloidosis has a poor prognosis and requires frequent follow-up in reference centres.
D. Guijarro, A. Jobbe-Duval, S. Aguilhon, F. Bauer, E. Donal, J. C. Eicher, J. Jeanneteau, B. Gellen, D. Kenizou, O. Lairez, B. Lequeux, D. Legallois, P. Réant, M. Salvat, M. F. Seronde, M. Kharoubi, A. Whereat, A. Farrugia, C. Taieb, A. Zaroui, T. Damy   +20 more
doaj   +1 more source

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

, 2018
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 forms of the serum carrier protein transthyretin (TTR), synthesized ...
Benson, Merrill D., Chung, Kevin, Cohn, Whitaker, Eisenberg, David S., Lee, Ji H., Saelices, Lorena, Whitelegge, Julian P.   +6 more
core   +1 more source

A phenomap of TTR amyloidosis to aid diagnostic screening

ESC Heart Failure, Volume 12, Issue 2, Page 1113-1118, April 2025.
Abstract Cardiac amyloidosis due to transthyretin (ATTR) remains an underdiagnosed cause of cardiomyopathy. As awareness of the disease grows and referrals for ATTR increase, clinicians are likely to encounter more atypical forms of the condition in clinical practice.
Alexios S. Antonopoulos, Theodoros Tsampras, George Lazaros, Konstantinos Tsioufis, Charalambos Vlachopoulos   +4 more
wiley   +1 more source

Oligonucleotide Drugs for Transthyretin Amyloidosis [PDF]

New England Journal of Medicine, 2018
In this issue of the Journal, Adams et al.1 and Benson et al.2 report the results of two randomized, double-blind, controlled trials testing the therapeutic efficacy of two different chemically mod...
Adams, D, Hawkins, PN, Polydefkis, M
openaire   +6 more sources

Misdiagnosis of hereditary amyloidosis as AL (Primary) amyloidosis [PDF]

, 2002
Background: Hereditary, autosomal dominant amyloidosis, caused by mutations in the genes encoding transthyretin, fibrinogen A -chain, lysozyme, or apolipoprotein A-I, is thought to be extremely rare and is not routinely included in the differential ...
Booth, D.R., Booth, S.E., Bybee, A., Gilbertson, J.A., Gillmore, J.D., Hawkins, P.N., Lachmann, H.J., Pepys, M.B.   +7 more
core  

Exercise limitations in amyloid cardiomyopathy assessed by cardiopulmonary exercise testing—A multicentre study

ESC Heart Failure, Volume 12, Issue 2, Page 1326-1335, April 2025.
Abstract Aims Amyloid cardiomyopathy is caused by the deposition of light chain (AL) or transthyretin amyloid (ATTR) fibrils, that leads to a restrictive cardiomyopathy, often resulting in heart failure (HF) with preserved or reduced ejection fraction.
Robin Willixhofer, Mauro Contini, Michele Emdin, Damiano Magrì, Alice Bonomi, Elisabetta Salvioni, Fabrizio Celeste, Alberico Del Torto, Claudio Passino, Christophe D.J. Capelle, Chiara Arzilli, Emiliano Fiori, Nicolò Capra, Christina Kronberger, Nikita Ermolaev, Andreas Kammerlander, Beatrice Musumeci, Giuseppe Vergaro, Vincenzo Castiglione, René Rettl, Giacomo Tini, Andrea Baggiano, Iacopo Fabiani, Susanna Sciomer, Roza Badr Eslam, Piergiuseppe Agostoni   +25 more
wiley   +1 more source

Molecular Tweezers Targeting Transthyretin Amyloidosis [PDF]

Neurotherapeutics, 2014
Transthyretin (TTR) amyloidoses comprise a wide spectrum of acquired and hereditary diseases triggered by extracellular deposition of toxic TTR aggregates in various organs. Despite recent advances regarding the elucidation of the molecular mechanisms underlying TTR misfolding and pathogenic self-assembly, there is still no effective therapy for ...
Ferreira, Nelson, Pereira-Henriques, Alda, Attar, Aida, Klärner, Frank-Gerrit, Schrader, Thomas, Gales, Luís, Saraiva, Maria, Almeida, Maria, Bitan, Gal   +8 more
openaire   +6 more sources

Negative for AL and ATTR: Could It Still Be Amyloid?

Annals of Internal Medicine: Clinical Cases
Amyloidosis is a systemic disease characterized by the extracellular deposition of misfolded proteins. We present a rare case of familial amyloidosis with multiorgan involvement.
Ahmed B. Elawad, Paul Wilkinson, Ryan Kimball, Ahmed F. Abdulrahim   +3 more
doaj   +1 more source

Gateway and journey of patients with cardiac amyloidosis

ESC Heart Failure, 2020
Aims Advances have been made over the last decade in the management of cardiac amyloidosis (CA), but a delayed diagnosis is still common. The aim of this study was to describe the journey to CA diagnosis from initial clinical and to analyse time to ...
Daniel Dang, Pauline Fournier, Eve Cariou, Antoine Huart, David Ribes, Pascal Cintas, Murielle Roussel, Magali Colombat, Yoan Lavie‐Badie, Didier Carrié, Michel Galinier, Olivier Lairez   +11 more
doaj   +1 more source