Abstract
Background Tafamidis is a transthyretin (TTR) stabilizer able to prevent mutated TTR tetramer dissociation into amyloidogenic monomers. There have been a few encouraging studies on safety and long-term efficacy of Tafamidis in early-onset Val30Met TTR-familial amyloid polyneuropathy (TTR-FAP) patients. However, less is known about its efficacy in later stages of the disease and in non-Val30Met mutations.
Highlights
Open AccessMonitoring safety and effectiveness of Tafamidis in transthyretin amyloidosis in Italy: a 3-year longitudinal multicenter study in a non-endemic area
Tafamidis is a transthyretin (TTR) stabilizer able to prevent mutated TTR tetramer dissociation into amyloidogenic monomers
Neuropathy and cardiomyopathy progressed in a significant proportion of patients despite treatment
Summary
Monitoring safety and effectiveness of Tafamidis in transthyretin amyloidosis in Italy: a 3-year longitudinal multicenter study in a non-endemic area. Andrea Cortese1*, Giuseppe Vita, Laura Obici, Marco Sabatelli, Gian Maria Fabrizi, Angelo Schenone, Giampaolo Merlini, Davide Pareyson. From First European Congress on Hereditary ATTR amyloidosis Paris, France. From First European Congress on Hereditary ATTR amyloidosis Paris, France. 2-3 November 2015
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