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Published in 2022 at "Journal of Neurology"
DOI: 10.1007/s00415-022-10978-3
Abstract: Hereditary amyloidosis with autosomal-dominant mutations in the transthyretin-gene (TTR) represents a severe multisystem disorder. Treatment options are now available for TTR familial amyloid polyneuropathy (TTR-FAP): patisiran [1], inotersen [2], tafamidis [3] and orthotopic liver transplantation…
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Keywords:
therapy;
tafamidis;
ttr;
amyloidosis ... See more keywords
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Published in 2020 at "Clinical therapeutics"
DOI: 10.1016/j.clinthera.2020.07.001
Abstract: PURPOSE An all-case, single-arm, observational, postmarketing surveillance is underway to assess the safety of tafamidis in patients with hereditary transthyretin (ATTRv) amyloidosis with peripheral polyneuropathy, also called transthyretin-type familial amyloid polyneuropathy, in Japan. Results from…
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Keywords:
safety;
interim analysis;
case;
amyloidosis ... See more keywords
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Published in 2020 at "European Heart Journal"
DOI: 10.1093/ehjci/ehaa946.2115
Abstract: In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis was shown to be an effective treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Further assessment of the efficacy of tafamidis in patients with more…
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Keywords:
patients advanced;
tafamidis patients;
class iii;
tafamidis ... See more keywords
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Published in 2020 at "Nephrology Dialysis Transplantation"
DOI: 10.1093/ndt/gfaa142.p0052
Abstract: Transthyretin (TTR) amyloidosis is a disease characterized by destabilization of the native TTR tetramer. It can be caused by aging or due to pathogenic mutations, and its diagnosis is often missed. Hereditary transthyretin amyloidosis (ATTRv),…
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Keywords:
administration;
time;
disease;
amyloidosis ... See more keywords
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Published in 2025 at "Circulation"
DOI: 10.1161/circ.152.suppl_3.4368953
Abstract: Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is an under-diagnosed and life-threatening progressive infiltrative cardiomyopathy characterized by the abnormal deposition of misfolded transthyretin (TTR) protein in the myocardium. The therapeutic options have traditionally been limited. Tafamidis is…
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Keywords:
transthyretin;
tafamidis;
review;
amyloid cardiomyopathy ... See more keywords
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Published in 2019 at "JCI insight"
DOI: 10.1172/jci.insight.126526
Abstract: BACKGROUNDThe hereditary transthyretin (TTR) amyloidoses are a group of diseases for which several disease-modifying treatments are now available. Long-term effectiveness of these therapies is not yet fully known. Moreover, the existence of alternative therapies has…
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Keywords:
response;
predictive model;
response tafamidis;
tafamidis ... See more keywords
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Published in 2019 at "Annals of Pharmacotherapy"
DOI: 10.1177/1060028019888489
Abstract: Objective: To review the pharmacology, efficacy, and safety of the selective transthyretin inhibitor tafamidis for transthyretin amyloid cardiomyopathy (ATTR-CM). Data Sources: A PubMed (1966 to October 2019) and ClinicalTrials. gov search was conducted using the…
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Keywords:
amyloid cardiomyopathy;
tafamidis first;
tafamidis;
pharmacology ... See more keywords
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Published in 2022 at "Hellenic journal of nuclear medicine"
DOI: 10.1967/s002449912483
Abstract: OBJECTIVE Cardiomyopathy is a common manifestation of transthyretin amyloidosis (ATTR), leading to heart failure, associated with high morbidity and mortality. The aim of this study was to investigate the effect of Tafamidis treatment by means…
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Keywords:
tafamidis;
method assessing;
tafamidis therapy;
response tafamidis ... See more keywords
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Published in 2023 at "Future cardiology"
DOI: 10.2217/fca-2022-0096
Abstract: WHAT IS THIS PLAIN LANGUAGE SUMMARY ABOUT? This summary presents the results from an ongoing, long-term extension study that followed an earlier study called ATTR-ACT. People who took part in this extension study and ATTR-ACT…
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Keywords:
long term;
tafamidis;
extension study;
attr act ... See more keywords