I’m not going to recap the 30+ hours of presentations from this year’s Conference. We will soon post many of the presentations and Sharon will blog about various presentations and projects over the next weeks and months. I wanted to tell you a bit about what took place this year and look forward, to share some thoughts about next year… Already you ask? We spend a lot of time thinking about the Conference, trying to improve it each year, trying to make sure we provide comprehensive information to all stakeholders and working on all fronts in order to accelerate treatments that will stop the progression of the disease for all boys everywhere, that will end Duchenne. We have not yet decided on the specifics as in date/location, but we have had some conversations about the nuts and bolts.
Pat Moeschen’s keynote was amazing. Everyone attending the meeting jammed into the room and for the first time ever, no cell phone rang, no one was texting. We were all listening to Pat. I would like to say, Pat is the voice of reason, but he is more than that. Pat is the voice of joy, of laughter, of living a life. Both the expert and sibling panel were amazing as well, with full audience participation. It confirmed my belief that the voice of this community needs to be heard. Parents, Patients, Siblings. And that when this voice is raised, people listen. We will continue to raise these voices next year and the year after that and the year after that.
Breakout sessions. Caring for an individual with Duchenne is difficult and often requires decisions that we (as parents) never imagined we would be faced with. Spending time in small sessions, expressing concern, asking questions, learning from experts, assure all of us we are doing our best and more than that, we motivate researchers and clinicians to think out of the box, to understand this critical need. Amanda Becker said it exactly right in the breakout session on Ataluren. One father mentioned his son’s ejection fraction dropped 20% after discontinuing Ataluren. While there is no data to confirm a direct relationship, stopping Ataluren was the only change to his son’s regimen. As each person expressed their own experience with Ataluren and interest in participating in an access program, Amanda said that in her view any change, any loss of function and surely a change in the ejection fraction constitutes an emergency. PTC’s medical officer had never thought of Duchenne in that way. Duchenne is an emergency.
Duchenne Therapeutics Development Meeting (aka –scientific track). The room was packed and the talks intense, hopeful, promising. Some parents came away shaking their head and suggesting they were able to understand about 20% of what was said, but very thankful to have the opportunity to attend.
Researchers/Clinicians agreed the meeting was successful, that a Duchenne Therapeutics meeting is important, but said they felt like they missed important sessions with parents. Parents had to choose and often felt torn, feeling the need to stay informed, but missing out on critical discussions relevant to their son’s life today. Next year’s plan will definitely include the Duchenne Therapeutics Development Meeting. We are exploring ideas about how to juggle things around a bit, which probably means adding more time to the meeting. Stay tuned.
Conference sessions felt nearly perfect – okay, I am biased. The early breakfast with Industry was informative, on both sides, as companies shared their experiences and addressed concerns. The plenary session included a broad range of topics, at first general (biomarkers, drug development, clinical trial development, multi-system trials, outcome measures) to very specific strategies such as exon skipping, utrophin, Lam iii, stem cells and viral gene therapy, and various cellular pathways involved in muscle degeneration.
The Levin family threw a lovely barbecue on Friday and by the Saturday gala, I think we were all a little foggy. The NJ staff had (nearly) killed themselves planning, preparing, and executing the Conference. Our brains were full of research, clinical care, friendships made and renewed…and HOPE. We awarded this year’s fellowship to Nick Dobes, who described his relationship with his friend Brian and one more time, we were reminded that our sons’ lives have a ripple effect throughout the community and the world and that all of us play a major role in advancing treatments. It was always obvious to me, but even more apparent when we are together, when we speak in ONE VOICE, we know we will change the world.
Looking forward to next year…