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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
Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides
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
Antisense inhibition of apolipoprotein (a) to lower plasma lipoprotein (a) levels in humans
Epidemiological, genetic association, and Mendelian randomization studies have provided strong evidence that lipoprotein (a) [Lp… Expand
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2016
2016
Interim Results of a Phase 2 Clinical Study of Nusinersen (ISIS-SMNRx) in Patients with Infantile-Onset Spinal Muscular Atrophy (P5.004)
Objective:To assess the safety, tolerability, pharmacokinetics and clinical effects of nusinersen (ISIS-SMNRx) in patients with… Expand
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2014
2014
Spinal Muscular Atrophy: Therapeutic Strategies
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
Characterization of a thermal neutron beam monitor based on gas electron multiplier technology Gabriele Croci1,4,∗, Carlo… Expand
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2014
2014
G.O.17: Results of a phase 2 open-label study of ISIS-SMNRx in patients with infantile (Type 1) spinal muscular atrophy
This study was conducted to evaluate the safety, tolerability, pharmacokinetics, and clinical efficacy of multiple intrathecal… Expand
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2014
2014
G.O.18: Results of a phase 2 study of ISIS-SMNRx in children with spinal muscular atrophy
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
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… Expand
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2007
2007
Spectral Coherence Interferometry (SCI) for fast and rugged industrial applications
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
New cytotoxic sesquiterpenes from the gorgonian Isis hippuris.
Five new suberosane sesquiterpenes, suberosenol A (1), suberosenol B (2), suberosanone (3), suberosenol A acetate (4), and… Expand
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