eteplirsen

Known as: (P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G),5'-(P-(4-((2-(2-(2-hydroxyethoxy)ethoxy)ethoxy)carbonyl)-1-piperazinyl)-N,N-dimethylphosphonamidate) RNA 
 
National Institutes of Health

Topic mentions per year

Topic mentions per year

2011-2017
0102020112017

Papers overview

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Review
2017
Review
2017
Eteplirsen, a phosphorodiamidate morpholino antisense oligonucleotide (PMO) that modulates splicing to treat Duchenne muscular… (More)
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2017
2017
BACKGROUND Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle… (More)
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2017
2017
W ith the food and drug administration (FDA) approval of nusinersen (trade name Spinraza) for treatment of spinal muscular… (More)
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Review
2017
Review
2017
INTRODUCTION In 2016 the US Food and Drug Administration (FDA) granted the marketing authorization for eteplirsen for Duchenne… (More)
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2017
2017
Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused by mutations in the dystrophin (DMD) gene… (More)
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Highly Cited
2016
Highly Cited
2016
OBJECTIVE To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer… (More)
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2016
2016
approval for eteplirsen by the FDA has introduced an exciting and potentially transformative era for Duchenne muscular dystrophy… (More)
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Review
2016
Review
2016
Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce exon 51 skipping that is developed by Sarepta… (More)
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Review
2016
Review
2016
This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are… (More)
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Review
2013
Review
2013
Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in 3,500-5,000 male births that is… (More)
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