|
rdf:type |
|
|
lifeskim:mentions |
|
|
pubmed:issue |
9791
|
|
pubmed:dateCreated |
2011-8-15
|
|
pubmed:databankReference |
|
|
pubmed:abstractText |
We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer (PMO) in patients with Duchenne muscular dystrophy.
|
|
pubmed:grant |
|
|
pubmed:commentsCorrections |
|
|
pubmed:language |
eng
|
|
pubmed:journal |
|
|
pubmed:citationSubset |
AIM
|
|
pubmed:chemical |
|
|
pubmed:status |
MEDLINE
|
|
pubmed:month |
Aug
|
|
pubmed:issn |
1474-547X
|
|
pubmed:author |
pubmed-author:AbbsStephenS,
pubmed-author:AnthonyKarenK,
pubmed-author:Arechavala-GomezaVirginiaV,
pubmed-author:BourkeJohnJ,
pubmed-author:BushbyKateK,
pubmed-author:CirakSebahattinS,
pubmed-author:DicksonGeorgeG,
pubmed-author:FengLucyL,
pubmed-author:GarraldaMaria ElenaME,
pubmed-author:GuglieriMichelaM,
pubmed-author:KoleRyszardR,
pubmed-author:MorganJennifer EJE,
pubmed-author:MuntoniFrancescoF,
pubmed-author:SewryCarolineC,
pubmed-author:ShrewsburyStephen BSB,
pubmed-author:StraubVolkerV,
pubmed-author:TorelliSilviaS,
pubmed-author:WellsDominic JDJ,
pubmed-author:WiltonSteve DSD,
pubmed-author:WoodMatthew J AMJ
|
|
pubmed:copyrightInfo |
Copyright © 2011 Elsevier Ltd. All rights reserved.
|
|
pubmed:issnType |
Electronic
|
|
pubmed:day |
13
|
|
pubmed:volume |
378
|
|
pubmed:owner |
NLM
|
|
pubmed:authorsComplete |
Y
|
|
pubmed:pagination |
595-605
|
|
pubmed:dateRevised |
2011-11-17
|
|
pubmed:meshHeading |
pubmed-meshheading:21784508-Adolescent,
pubmed-meshheading:21784508-Alternative Splicing,
pubmed-meshheading:21784508-Child,
pubmed-meshheading:21784508-Dose-Response Relationship, Drug,
pubmed-meshheading:21784508-Dystrophin,
pubmed-meshheading:21784508-Exons,
pubmed-meshheading:21784508-Humans,
pubmed-meshheading:21784508-Infusions, Intravenous,
pubmed-meshheading:21784508-Male,
pubmed-meshheading:21784508-Morpholines,
pubmed-meshheading:21784508-Morpholinos,
pubmed-meshheading:21784508-Muscle, Skeletal,
pubmed-meshheading:21784508-Muscular Dystrophy, Duchenne,
pubmed-meshheading:21784508-Oligonucleotides
|
|
pubmed:year |
2011
|
|
pubmed:articleTitle |
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
|
|
pubmed:affiliation |
Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK.
|
|
pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't,
Multicenter Study,
Clinical Trial, Phase II
|
