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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediate...

Adams, David; Polydefkis, Michael; González-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt V.; Schmidt, Hartmut H.; Berk, John L.

Background: Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Methods: This multicentre, open-label ex...


European Society of Cardiology : cardiovascular disease statistics 2017

Timmis, Adam; Townsend, Nick; Gale, Chris; Grobbee, Rick; Maniadakis, Nikos; Flather, Marcus; Wilkins, Elizabeth; Wright, Lucy; Vos, Rimke; Bax, Jeroen

Aims: The European Society of Cardiology (ESC) Atlas has been compiled by the European Heart Agency to document cardiovascular disease (CVD) statistics of the 56 ESC member countries. A major aim of this 2017 data presentation has been to compare high-income and middle-income ESC member countries to identify inequalities in disease burden, outcomes, and service provision. Methods and results: The Atlas utilizes...


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