Results 51 to 60 of about 6,309 (194)

Analysis of post-market adverse events of tafamidis base on the FDA adverse event reporting system

Scientific Reports
Tafamidis is the world's first and only oral drug approved to treat the rare disease transthyretin amyloid cardiomyopathy (ATTR-CM). Medicines are known to have different adverse reactions during the course of treatment.
Fan Wu, He Zhu, Yue Zhang
doaj   +1 more source

Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy

Clinical Medicine Insights: Cardiology, 2017
Background: Transthyretin cardiomyopathy (TTR-CM) is a progressive, fatal disease caused by the accumulation of misfolded transthyretin (TTR) amyloid fibrils in the heart.
Marla B Sultan, Balarama Gundapaneni, Jennifer Schumacher, Jeffrey H Schwartz   +3 more
doaj   +1 more source

Inhibition by small-molecule ligands of formation of amyloid fibrils of an immunoglobulin light chain variable domain. [PDF]

, 2015
Overproduction of immunoglobulin light chains leads to systemic amyloidosis, a lethal disease characterized by the formation of amyloid fibrils in patients' tissues. Excess light chains are in equilibrium between dimers and less stable monomers which can
Brumshtein, Boris, Cascio, Duilio, Eisenberg, David S, Esswein, Shannon R, Ly, Alan T, Phillips, Martin L, Salwinski, Lukasz, Sawaya, Michael R   +7 more
core   +1 more source

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy [PDF]

New England Journal of Medicine, 2018
Transthyretin amyloid cardiomyopathy is caused by the deposition of transthyretin amyloid fibrils in the myocardium. The deposition occurs when wild-type or variant transthyretin becomes unstable and misfolds. Tafamidis binds to transthyretin, preventing tetramer dissociation and amyloidogenesis.In a multicenter, international, double-blind, placebo ...
Maurer, M. S., Schwartz, J. H., Gundapaneni, B., Elliott, P. M., Merlini, G., Waddington-Cruz, M., Kristen, A. V., Grogan, M., Witteles, R., Damy, T., Drachman, B. M., Shah, S. J., Hanna, M., Judge, D. P., Barsdorf, A. I., Huber, P., Patterson, T. A., Riley, S., Schumacher, J., Stewart, M., Sultan, M. B., Rapezzi, C., Investigators, Attr-Act Study   +22 more
openaire   +4 more sources

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

Updated Evaluation of the Safety, Efficacy and Tolerability of Tafamidis in the Treatment of Hereditary Transthyretin Amyloid Polyneuropathy

Drug, Healthcare and Patient Safety, 2023
Catarina Falcão de Campos,1,2 Isabel Conceição1,2 1Department of Neurosciences and Mental Health, Centro Hospitalar Universitário de Lisboa-Norte, Lisbon, Portugal; 2Instituto de Fisiologia, Instituto de Medicina Molecular, Centro de Estudos Egas Moniz ...
Falcão de Campos C, Conceição I
doaj  

A woman with a rare p.Glu74Gly transthyretin mutation presenting exclusively with a rapidly progressive neuropathy: a case report [PDF]

, 2014
Introduction: Familial amyloid polyneuropathy is a rare autosomal dominant disorder caused by mutations in the transthyretin gene, TTR. Diagnosis can be challenging, especially if other family members are not affected or an obvious systemic involvement ...
Acker, Till, Haverkamp, Thomas, Kimmich, Christoph, Krämer, Heidrun H., Röcken, Christoph, Schänzer, Anne, Weidner, Isabell   +6 more
core   +1 more source

Negative [99mTc]Tc ‐DPD Scintigraphy, Presence of Monoclonal Protein and Biopsy Suggestive of AL Amyloidosis in a Patient With Homozygous p.Ala101Val Transthyretin Gene Variant

Clinical Case Reports, Volume 14, Issue 3, March 2026.
ABSTRACT Amyloidosis is a rare disease associated with the deposition of misfolded proteins that damage multiple organs, leading to a wide range of symptoms. The most frequently implicated proteins in amyloidosis include immunoglobulin Free Light Chains (FLC), related to AL amyloidosis, and transthyretin (TTR), which is responsible for ATTR amyloidosis.
Paulina Kryszpin, Piotr Jachimowski, Łukasz Augustowski, Mateusz Ziarkiewicz, Grzegorz Basak, Marta Lipowska, Marta Legatowicz‐Koprowska, Bogna Ziarkiewicz‐Wróblewska, Monika Gawor‐Prokopczyk   +8 more
wiley   +1 more source

Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients:an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies [PDF]

, 2021
AIM: The Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) showed that tafamidis reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
Bhambri, Rahul, Garcia, Andrea, Grima, Daniel, Heeg, Bart, Li, Benjamin, Masri, Ahmad, Postma, Maarten, Rozenbaum, Mark H., Stewart, Michelle, Tran, Diana   +9 more
core   +1 more source

Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin‐Mediated Amyloidosis: A Systematic Review

Health Science Reports, Volume 9, Issue 3, March 2026.
ABSTRACT Background Hereditary transthyretin‐mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life‐threatening conditions like cardiomyopathy and polyneuropathy. For treatment of hATTR in the new medical realm, there is a breakthrough discovery of a medicine called Eplontersen, which targets the underlying ...
Zeel Vishnubhai Patel, Verkha Kumari, Paras, Bethineedi Lakshmi Deepak, Priyadarshi Prajjwal, Kolanu Nikhil Deep, Rakhi Bai, Serene Sara John, Omniat Amir Hussin   +8 more
wiley   +1 more source