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Background Transthyretin (TTR) amyloidosis is a progressive and fatal systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the heart, peripheral nerves and other organ systems. TTR Amyloidosis-associated cardiomyopathy (ATTR-CM), caused by TTR amyloid infiltration of the myocardium and conduction system, results in a restrictive cardiomyopathy associated with atrial arrhythmias, progressive heart failure with preserved ejection fraction in early phases, and leads to reduced life expectancy. ISIS-TTRRx is a first-in-class, 2nd-generation antisense oligonucleotide designed to produce substantial reductions in the levels of both mutant and wild type TTR produced from the liver.

orphanet journal of rare diseases

A phase 3 clinical trial with ISIS-TTRRx, a 2nd-generation antisense oligonucleotide targeting transthyretin (TTR), for the treatment of TTR amyloid cardiomyopathy