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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

Papers overview

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2019
2019
Background: Duchenne muscular dystrophy (DMD) patients experience skeletal muscle degeneration, including respiratory muscles… Expand
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2018
2018
Objective To describe the quantification of novel dystrophin production in patients with Duchenne muscular dystrophy (DMD) after… Expand
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2018
2018
BACKGROUND Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle… Expand
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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… Expand
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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… Expand
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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… Expand
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2016
2016
New therapies are much needed for Duchenne muscular dystrophy. Recent data from a phase II clinical trial has led to accelerated… Expand
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Highly Cited
2013
Highly Cited
2013
OBJECTIVE In prior open-label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in… Expand
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Highly Cited
2013
Highly Cited
2013
Duchenne muscular Dystrophy (DMD) is an inherited disease caused by mutations in the dystrophin gene that disrupt the open… Expand
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Highly Cited
2012
Highly Cited
2012
We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the… Expand
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