FAQ

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… (More)
National Institutes of Health

Topic mentions per year

Topic mentions per year

1998-2016
024619982016

Papers overview

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Highly Cited
2016
Highly Cited
2016
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
OBJECTIVE To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen… (More)
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2016
2016
Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides.
Transthyretin amyloidosis (ATTR amyloidosis) is a rare disease that results from the deposition of misfolded transthyretin (TTR… (More)
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2016
2016
Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells
Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, predominantly expressed in the… (More)
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Highly Cited
2015
Highly Cited
2015
Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study.
BACKGROUND Lipoprotein(a) (Lp[a]) is a risk factor for cardiovascular disease and calcific aortic valve stenosis. No effective… (More)
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2015
2015
Results of a proof of concept, double-blind, randomized trial of a second generation antisense oligonucleotide targeting high-sensitivity C-reactive protein (hs-CRP) in rheumatoid arthritis
INTRODUCTION This randomized, double-blind, phase II study evaluated the pharmacodynamics, safety and tolerability of ISIS 329993… (More)
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2015
2015
The effect of C-reactive protein reduction with a highly specific antisense oligonucleotide on atrial fibrillation assessed using beat-to-beat pacemaker Holter follow-up
C-reactive protein (CRP) is known to be strongly associated with atrial fibrillation (AF). However, it is not clear if CRP is a… (More)
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2014
2014
Effects of an Antisense Oligonucleotide Inhibitor of C‐Reactive Protein Synthesis on the Endotoxin Challenge Response in Healthy Human Male Volunteers
BACKGROUND C-reactive protein (CRP) binds to damaged cells, activates the classical complement pathway, is elevated in multiple… (More)
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Highly Cited
2012
Highly Cited
2012
Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy.
Transthyretin (TTR)-associated amyloidosis is a late-onset autosomal-dominant genetic disease. Over 100 amyloidogenic mutations… (More)
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2011
2011
Four new polyoxygenated gorgosterols from the gorgonian Isis hippuris.
Four new polyoxygenated steroids (1-4) together with four known ones (5-8) have been isolated from the gorgonian Isis hippuris… (More)
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2005
2005
Polyoxygenated steroids from the gorgonian Isis hippuris.
Eleven new hippuristanols (1-4, 7-9, and 11-14), along with eight known metabolites (5, 6, 10, and 15-19), have been isolated… (More)
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