The European Medicine Agency (EMA) hosted a workshop on developing exon skipping therapies for Duchenne muscular dystrophy, this was organized by this COST Action with the SCOPE-DMD project. The meeting took place on 29 April 2015 and was a follow up to the “Antisense oligonucleotide therapy development for Duchenne muscular dystrophy“ meeting which was organized by TREAT-NMD and hosted by EMA in 2009.
The workshop was an open forum discussion on the regulatory and translational challenges of developing exon skipping therapies for Duchenne and involved patient representatives, regulators, academics, clinicians and industry representatives.
Monica Ensini: ‘This stakeholder meeting was a great example of how you can move things forward, in a constructive, non-confrontational manner. The meeting was of very high quality, based on science and data provided by each of the stakeholders present.’