194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8-10 December 2012, Naarden, The Netherlands

摘要

Twenty-seven participants from 9 countries (Australia; Belgium; England; France; Germany; Italy; Japan; The Netherlands; USA) attended the third ENMC workshop on exon skipping "Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy." The topic of this workshop was on 'Streamlining the development path of exon skipping compounds' and followed the formula of similar workshops held in 2004 on 'Antisense oligonucleotides in DMD', which focused on intramuscular administration of antisense oligonucleotides and in 2007 on 'Planning Phase I/II Clinical Trials using systemically delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy (DMD)'.