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Transthyretin Antisense Oligonucleotide ISIS-TTR Rx

Known as: ISIS-TTR Rx, ISIS-TTRRx, TTR ASO ISIS-TTR Rx 
An antisense oligonucleotide (ASO) targeting transthyretin (TTR), which has potential use in the treatment of TTR amyloidosis (ATTR). Upon… 
National Institutes of Health

Papers overview

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Review
2016
Review
2016
Epidemiological, genetic association, and Mendelian randomization studies have provided strong evidence that lipoprotein (a) [Lp… 
Highly Cited
2016
Highly Cited
2016
Abstract Transthyretin amyloidosis (ATTR amyloidosis) is a rare disease that results from the deposition of misfolded… 
2015
2015
Objective: A Phase 3 randomized, double-blind, placebo-controlled study to assess the efficacy and safety of ISIS-TTR Rx in… 
2014
2014
Opinion statementSpinal muscular atrophy is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the… 
2014
2014
Characterization of a thermal neutron beam monitor based on gas electron multiplier technology Gabriele Croci1,4,∗, Carlo… 
Highly Cited
2012
Highly Cited
2012
Transthyretin (TTR)-associated amyloidosis is a late-onset autosomal-dominant genetic disease. Over 100 amyloidogenic mutations… 
2000
2000
Five new suberosane sesquiterpenes, suberosenol A (1), suberosenol B (2), suberosanone (3), suberosenol A acetate (4), and…