|1.||Feng, Lucy: 3 articles (02/2012 - 10/2009)|
|2.||Sewry, Caroline: 3 articles (02/2012 - 10/2009)|
|3.||Morgan, Jennifer E: 3 articles (02/2012 - 10/2009)|
|4.||Muntoni, Francesco: 3 articles (02/2012 - 10/2009)|
|5.||Arechavala-Gomeza, Virginia: 3 articles (02/2012 - 10/2009)|
|6.||Cirak, Sebahattin: 3 articles (02/2012 - 10/2009)|
|7.||Torelli, Silvia: 2 articles (02/2012 - 08/2011)|
|8.||Anthony, Karen: 2 articles (02/2012 - 08/2011)|
|9.||Wells, Dominic J: 2 articles (08/2011 - 10/2009)|
|10.||Abbs, Stephen: 2 articles (08/2011 - 10/2009)|
|1.||Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker)
08/13/2011 - "The safety and biochemical efficacy that we present show the potential of AVI-4658 to become a disease-modifying drug for Duchenne muscular dystrophy. "
10/01/2009 - "Seven patients with Duchenne muscular dystrophy with deletions in the open reading frame of DMD that are responsive to exon 51 skipping were selected on the basis of the preservation of their extensor digitorum brevis (EDB) muscle seen on MRI and the response of cultured fibroblasts from a skin biopsy to AVI-4658. "
02/01/2012 - "We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the morpholino splice-switching oligonucleotide AVI-4658 (eteplirsen) that induces skipping of dystrophin exon 51 in patients with relevant deletions, restores the open reading frame and induces dystrophin protein expression after intramuscular (i.m.) injection. "
08/13/2011 - "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. "
10/01/2009 - "Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study."
|2.||phosphorodiamidate morpholino oligomer