128th ENMC International Workshop on ‘Preclinical optimization and Phase I/II Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy’ 22–24 October 2004, Naarden, The Netherlands

Abstract

Twenty-six participants including parents, scientists, industry representatives and clinicians from Australia, Belgium, England, France, The Netherlands, and USA attended the 128th ENMC workshop on the topic of ‘Preclinical optimization and Phase I/II Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy’. The meeting was held in Naarden, The Netherlands, during the weekend of the 22nd–24th October 2004. The aim of the meeting was for the two European consortia (one in The Netherlands/Belgium and the other in the UK) that are preparing for a clinical trial on antisense oligonucleotides (AON) in Duchenne Muscular Dystrophy (DMD) to meet and compare their respective protocols. In addition, the meeting was attended by experts on AON in DMD from other countries and by experts on the use of AON in other fields of medicine. Representatives of four companies, ISIS Pharmaceuticals, Prosensa, Afforce Healthcare and Transgene attended the workshop as well, together with a parents representative. DMD is a common and severe form of muscular dystrophy, caused by intragenic mutations in the dystrophin gene. The majority of these mutations are out-of-frame deletions (and duplications); in-frame deletions/duplications characterize the milder allelic variant Becker muscular dystrophy (BMD).