caa a22 4500 605479224 CHVBK 20210128100409.0 cr unu---uuuuu 210128e20150301xx s 000 0 eng 10.1007/s10741-014-9462-7 doi (NATIONALLICENCE)springer-10.1007/s10741-014-9462-7 Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs [Elektronische Daten] [Adam Castaño, Brian Drachman, Daniel Judge, Mathew Maurer] Transthyretin-cardiac amyloidoses (ATTR-CA) are an underdiagnosed but increasingly recognized cause of heart failure. Extracellular deposition of fibrillary proteins into tissues due to a variety of inherited transthyretin mutations in ATTRm or due to advanced age in ATTRwt eventually leads to organ failure. In the heart, amyloid deposition causes diastolic dysfunction, restrictive cardiomyopathy with progressive loss of systolic function, arrhythmias, and heart failure. While traditional treatments have consisted of conventional heart failure management and supportive care for systemic symptoms, numerous disease-modifying therapies have emerged over the past decade. From organ transplantation to transthyretin stabilizers (diflunisal, tafamidis, AG-1), TTR silencers (ALN-ATTR02, ISIS-TTR(Rx)), and degraders of amyloid fibrils (doxycycline/TUDCA), the potential for effective transthyretin amyloid therapy is greater now than ever before. In light of these multiple agents under investigation in human clinical trials, clinicians should be familiar with the systemic cardiac amyloidoses, their differing pathophysiology, natural histories, and unique treatment strategies. Springer Science+Business Media New York, 2014 Cardiac amyloidosis nationallicence Transthyretin nationallicence Cardiomyopathy nationallicence Senile systemic amyloidosis nationallicence Familial amyloid polyneuropathy nationallicence Diflunisal nationallicence ALN-TTR02 nationallicence ALN-TTRSc nationallicence Tafamidis nationallicence Doxycycline nationallicence TUDCA nationallicence siRNA nationallicence Oligonucleotides nationallicence Castaño Adam Center for Advanced Cardiac Care, Columbia College of Physicians and Surgeons, New York City, NY, USA aut Drachman Brian Division of Cardiology, University of Pennsylvania, Philadelphia, PA, USA aut Judge Daniel Division of Cardiology, Johns Hopkins University, Baltimore, MD, USA aut Maurer Mathew Center for Advanced Cardiac Care, Columbia College of Physicians and Surgeons, New York City, NY, USA aut Heart Failure Reviews Springer US; http://www.springer-ny.com 20/2(2015-03-01), 163-178 1382-4147 20:2<163 2015 20 10741 https://doi.org/10.1007/s10741-014-9462-7 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s10741-014-9462-7 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Castaño Adam Center for Advanced Cardiac Care, Columbia College of Physicians and Surgeons, New York City, NY, USA aut NATIONALLICENCE 700 1- Drachman Brian Division of Cardiology, University of Pennsylvania, Philadelphia, PA, USA aut NATIONALLICENCE 700 1- Judge Daniel Division of Cardiology, Johns Hopkins University, Baltimore, MD, USA aut NATIONALLICENCE 700 1- Maurer Mathew Center for Advanced Cardiac Care, Columbia College of Physicians and Surgeons, New York City, NY, USA aut NATIONALLICENCE 773 0- Heart Failure Reviews Springer US; http://www.springer-ny.com 20/2(2015-03-01), 163-178 1382-4147 20:2<163 2015 20 10741