A phase 3 clinical trial with ISIS-TTRRx, a 2nd-generation antisense oligonucleotide targeting transthyretin (TTR), for the treatment of TTR amyloid cardiomyopathy
نویسندگان
چکیده
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.
منابع مشابه
Treatment of transthyretin (TTR) amyloid cardiomyopathy with an antisense oligonucleotide inhibitor of TTR synthesis
Background While transthyretin (TTR) amyloidosis is usually characterized by peripheral and autonomic neuropathy, a majority of patients also have evidence of restrictive cardiomyopathy. In addition, amyloid cardiomyopathy may occur in the elderly without the presence of a mutant form of TTR. Both the hereditary and wild-type TTR amyloidosis are characterized by progressive restrictive cardiomy...
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