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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… Expand
National Institutes of Health

Papers overview

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Highly Cited
2016
Highly Cited
2016
Abstract Transthyretin amyloidosis (ATTR amyloidosis) is a rare disease that results from the deposition of misfolded… Expand
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Review
2016
Review
2016
Epidemiological, genetic association, and Mendelian randomization studies have provided strong evidence that lipoprotein (a) [Lp… Expand
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2016
2016
Objective:To assess the safety, tolerability, pharmacokinetics and clinical effects of nusinersen (ISIS-SMNRx) in patients with… Expand
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2014
2014
Opinion statementSpinal muscular atrophy is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the… Expand
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2014
2014
Characterization of a thermal neutron beam monitor based on gas electron multiplier technology Gabriele Croci1,4,∗, Carlo… Expand
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2014
2014
This study was conducted to evaluate the safety, tolerability, pharmacokinetics, and clinical efficacy of multiple intrathecal… Expand
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2014
2014
This open-label, multiple ascending-dose study was conducted to evaluate the safety, tolerability, pharmacokinetics… Expand
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Highly Cited
2012
Highly Cited
2012
Transthyretin (TTR)-associated amyloidosis is a late-onset autosomal-dominant genetic disease. Over 100 amyloidogenic mutations… Expand
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2007
2007
Summary form only given. Standard optical coherence tomography (OCT) has been proven to provide surface and general 3D metrology… Expand
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2000
2000
Five new suberosane sesquiterpenes, suberosenol A (1), suberosenol B (2), suberosanone (3), suberosenol A acetate (4), and… Expand
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