Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
Author(s) -
Matthew J. Maurer,
Jeffrey H. Schwartz,
Balarama Gundapaneni,
Perry Elliott,
Giampaolo Merlini,
Márcia WaddingtonCruz,
Arnt V. Kristen,
Martha Grogan,
Ronald Witteles,
Thibaud Damy,
Brian Drachman,
Sanjiv J. Shah,
Mazen Hanna,
Daniel P. Judge,
Alexandra I. Barsdorf,
Peter M. Huber,
Terrell A. Patterson,
Steve Riley,
Jennifer Schumacher,
Michelle Stewart,
Marla B. Sultan,
Claudio Rapezzi
Publication year - 2018
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1805689
Subject(s) - transthyretin , hazard ratio , medicine , placebo , clinical endpoint , confidence interval , cardiomyopathy , gastroenterology , cardiology , clinical trial , heart failure , pathology , alternative medicine
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.
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