Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes.

@article{Maurer2015TafamidisIT,
  title={Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes.},
  author={Mathew S Maurer and Donna Roy Grogan and Daniel P Judge and Rajiv Mundayat and Jeff Packman and Ilise D Lombardo and Arshed A Quyyumi and Janske Aarts and Rodney Howard Falk},
  journal={Circulation. Heart failure},
  year={2015},
  volume={8 3},
  pages={519-26}
}
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 with respect to progression of TTR amyloid cardiomyopathy. Thirty-one wild-type… CONTINUE READING

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Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis • 2012

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade.

Proceedings of the National Academy of Sciences of the United States of America • 2012
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