Exon Skipping & Improving Industry Communications

The 194th workshop of the European Neuro Muscular Centre (ENMC) met in Naarden, The Netherlands, December 7-9, 2012. The title of the workshop was, “Towards Clinical Application of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy – Opportunities and Challenges.”  25 representatives of industry, research, care and advocacy from around the world attended the workshop. 


Industry and research presented the current state of the science of exon skipping: where trials stand and result so far.   Future directions, including the challenges of developing additional antisense oligonucleotides (AO’s), detecting and improving delivery of AO’s, responses to treatment were discussed. Potential outcome measures and initiatives for clinical trails in smaller subpopulations were discussed. Finally, the importance of affecting the cardiac muscle was stressed.


Parent and patient representatives from the US, The Netherlands and Italy discussed the importance of communication – accurate, precise, timely and transparent communication. The impact of social media, and the responsibility of all parties to be aware of messages that are given and received online was stressed. Everyone agreed that we are indeed in a new era of communication. Social media may not be recognized by industry and research as credible, but the ability of sites such as Facebook to impact a huge portion of the community in a very brief amount of time was recognized. The take home message was that parents and patients appreciate regular communication, both when there is and where there is not something new to report.


As a result of this meeting, a working group addressing communication was formed. The working group was charged with the task of developing a method for industry to give regular (quarterly) reports to patient/parent organization, which will then publish them for the Duchenne and Becker community. This will enable the Duchenne and Becker community to have available, timely access to accurate information, which comes straight from industry and is delivered in a consistent manner to the entire community. The working group will keep us updated as to their progress and hopes to have a working method of information in place within the next few months.


Update 1/16/13: Read the reports from the workshop

Kathi Kinnett, Director of Clinical Care
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