PLoS One
Publication date:
2016-01-01
Volume:
11
Publisher:
Public Library of Sciene
Author:
Goemans, Nathalie
Tulinius, Már ; Van den Hauwe, Marleen ; Kroksmark, Anna-Karin ; Buyse, Gunnar ; Wilson, Rosamund J ; van Deutekom, Judith C ; de Kimpe, Sjef J ; Lourbakos, Afrodite ; Campion, Giles
Keywords:
Science & Technology, Multidisciplinary Sciences, Science & Technology - Other Topics, 6-MINUTE WALK TEST, ANTISENSE OLIGONUCLEOTIDE, DISEASE PROGRESSION, EXPRESSION, DELETIONS, CHILDREN, Adolescent, Child, Child, Preschool, Dystrophin, Exercise Test, Humans, Male, Muscle, Skeletal, Muscular Dystrophy, Duchenne, Oligonucleotides, Treatment Outcome, Walking, General Science & Technology
Abstract:
Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.