I like the idea of New Year’s Resolutions. It makes perfect sense to look backwards for a moment, magnify the events in an effort to scrutinize what was done, learn from it, identify what might have been done better, with more finesse, more commitment, more strategic investment, more collaboration. And I always think about starting early, in December for instance. But in December, we put up the tree and think about the holidays. We keep the tree lights twinkling day and night and no matter what beliefs we hold as individuals, there is a special feeling about the holidays and interrupting them to consider Resolutions just does not feel quite right.
Our celebrations were slightly delayed as the snow interrupted everyone’s plans. Michelle arrived just before and Jenny arrived just after Christmas. Time ran out for Kris to make the trip.
We had a quiet celebration, a lovely restful few days of just hanging out together. New Year’s Resolutions were mentioned and quickly dismissed as we helped ourselves to sugar cookies and dark chocolate. On New Year’s Eve, Jenny and Michelle returned to New York and London. The tree no longer sparkled in anticipation of their visit. In fact, without them, the lights of Christmas had lost their luster and the tree had lost its fragrance.
As I took the tree down, I revisited the idea of New Years’ Resolutions, tiring as it seemed, and for just one moment, imagined that I was the one given the magic marker and some sort of assurance that whatever I wrote on the New Year’s wipe board would come true. I started with the usual, improve diet, exercise more –train for the upcoming Disney ½ marathon, drink less Diet Coke… suddenly taking off in the direction of what we, as a community, might hope for in 2011.
- One Voice Summit – Feb. 14, 2001 Senator Specter held a hearing on muscular dystrophy. Three different panels testified about the state of research and what might change if the MD-CARE Act would become law. In Dec. 2001, the MD-CARE Act became law and was re-authorized in 2008. We will focus on the Action Plan for the Muscular Dystrophies and discuss the impact of this legislation on our community when we meet next month. Please join us!
- Clarity around exon skipping – Prosensa/GSK; AVI focused on mutations in the range of 40-50. Several groups working on ‘accelerating’ exon skipping, working on rare mutations. But what mutations, what expertise, what timeline and how can we, as a community, work together to overcome existing barriers?
- Focus on the heart –accelerating Serca 2a, convene top thought leaders in pediatric cardiology with Duchenne experts, and give cardiac issues a greater sense of urgency.
- Utrophin – Cross fingers and pray, Project Catalyst’s utrophin up-regulation will take a leap forward – clinical candidate; Speaking with Summit as they reformulate C1100
- Acceleron/Shire will announce a Phase II for Ace -031
- Multi-centered trial /combined therapeutics – TBA 1st quarter
- Combined Therapeutics/Dog model
- Expand public/private partnerships with NIH
- Additional partnerships with Industry
- Working groups established from Endocrine meeting –focus on bone health, puberty, growth, and insulin metabolism
- Increased Awareness – It has been an amazing year for raising awareness in a broad range of audiences – books on Duchenne, The New Yorker piece on Duchenne, Coach to Cure MD and Cadillac’s PSA. Hopefully this will mean in 2011, that no parent in this community will hear the words “so your son has MS.”
At the end of the day, my New Year’s Resolution is to continue to work with this amazing, dedicated community, to make a difference in the lives of all of our young men (and women) with Duchenne because, in point of fact, being part of this community has made such a difference in my own life and I am very grateful.