Our Action in the media
You can now download the briefing document from the SCOPE-DMD & COST Action meeting, “Antisense oligonucleotide – mediated exon skipping therapy development for Duchenne muscular dystrophy (DMD)” which took place in April 2015 and was hosted by the European Medicines Agency. A policy review based on the collaborative efforts leading to this meeting is now published in Lancet Neurology, however videos of talks from the day are now available online.
Virginia Arechavala-Gomeza, Bernard Khoo and Anemieke Aartsma-Rus wrote a review on splicing modulation therapy for the treatment of genetic diseases. It also mentions the COST Action. You can download the paper here: http://www.dovepress.com/articles.php?article_id=19411.
COST Action members of the regulatory models and biochemical outcome measures working groups met on May 26 2014 in Leiden to discuss translational and regulatory challenges for clinical development of exon skipping. The meeting report has been published in Human Gene Therapy and can be found at http://www.ncbi.nlm.nih.gov/pubmed/25184444. A summary for the general public has been produced as well and can be found here.
Annemieke Aartsma-Rus was guest editor of a special issue of the journal Nucleic Acids Therapeutics on exon skipping, which contains contribution of multiple members of our Action.
The online version of this issue can be found here: http://online.liebertpub.com/toc/nat/24/1
The editorial mentioning our Action 1207 can be found here: http://online.liebertpub.com/doi/pdfplus/10.1089/nat.2014.1500
Dr Virginia Arechavala-Gomeza explaining exon skipping
Exon skipping dance
An explanation of exon skipping for Duchenne muscular dystrophy in danceform: www.dmd.nl/gt/dance
An explanation on biomarkers and biomarker development for DMD can be found here.
This COST Action and TREAT-NMD organized a workshop in London June 21st 2013 to discuss outstanding issues to come to a coordinated response to the public consultation by the EMA on the draft guidance for clinical investigation of medicinal products in BMD/DMD. A meeting report has been published in Neuromuscular disorders.