Results 61 to 70 of about 16,120 (219)

Liver Transplantation for Transthyretin Amyloidosis

, 2009
Liver transplantation has until now proved to be the only treatment available that halts the progression of hereditary transthyretin (TTR) associated amyloidosis.
Ole B. Suhr, Lundgren, E, Ericzon, B-G, Ericzon, Bo-Göran,, Erik Lundgren, Lundgren, Erik,, Bo-Göran Ericzon, Suhr, OB, Suhr, Ole B.,   +8 more
core   +1 more source

Ten questions about transthyretin amyloidosis

, 2022
Systemic amyloidosis is a hereditary or acquired disease characterized by deposition of amyloid insoluble fibrils into body organs and tissues, causing structural abnormalities and organ dysfunction, i.e. heart failure.
Gagliardi C., Galie N., Massa P., Guaraldi P., Caponetti A. G., Leone O., Biagini E., Saturi G., Longhi S., Sguazzotti M., Ponziani A., Giovannetti A., Cortelli P., Accietto A.   +13 more
core   +1 more source

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

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

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

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

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Helical reconstruction of amyloids in cryoSPARC

Acta Crystallographica Section F, EarlyView.
We present practical guidelines for helical reconstruction of amyloid filaments in cryoSPARC, benchmarked against publicly available datasets. Our analysis defines current capabilities and limitations, and outlines optimization strategies to enable high‐throughput structural studies and therapeutic discovery.Amyloid‐mediated proteotoxicity underlies ...
Jan-Hannes Schaefer, Robert T. O'Neill, Joseph P. Donnelly, Evan T. Powers, Jeffery W. Kelly, Gabriel C. Lander   +5 more
wiley   +1 more source

New Medications in the Treatment of Hereditary Transthyretin Amyloidosis

, 2018
Hereditary transthyretin amyloidosis is an inherited disorder that results in the gradual progressive deposit of abnormal protein called amyloid in the body’s organs and tissues.
Scot Walker
core   +1 more source