Article
Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes.
Circulation. Heart failure - 1 May 2015
Maurer Mathew S, Grogan Donna R, Judge Daniel P, Mundayat Rajiv, Packman Jeff, Lombardo Ilise, Quyyumi Arshed A, Aarts Janske, Falk Rodney H
Abstract excerpt
BACKGROUND: Transthyretin (TTR) amyloidosis is a progressive systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the heart and systemically as amyloid. METHODS AND RESULTS: This phase 2 open-label trial evaluated the stabilization of TTR tetramers using 20 mg of tafamidis daily at week 6 (primary end point), month 6, and month 12, as well as safety of tafamidis treatment and efficacy...
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