Cincinnati Conference
It's been three weeks since the DMD conference in Cincinnati, and I am still reeling from the onslaught of information. I was expecting an intense academic experience. I arrived with several notebooks, a list of questions for different scientists and physicians, and a plan for which breakout sessions to attend (with backups in case our first choices weren't helpful). Even with this preparation, my brain was still tied up in knots from the two long days of highly scientific presentations.
To look on the bright side (which I still try to do), it's good that there was so much to say. The hot topic of the weekend was definitely exon skipping. Several researchers presented their work in this area, and the general consensus was that exon skipping currently represents our best hope for children with DMD. That made us feel good about our decision to fund research in this area.
Many presenters discussed other approaches to a cure or treatment for DMD. Depressingly, some work is turning up dead ends. Stem cell research does not appear to be progressing toward a viable treatment for muscular dystrophy at this time. However, small molecule drug discovery and gene replacement therapy show serious potential. We plan to fund research into these areas in the near future.
One thing I was not prepared for was the emotional turmoil I experienced at the conference. I arrived at the meeting somewhat excited, bizarre as that may sound. I was with my husband on a child-free trip (except for baby Maisy, who is with me 24/7). We spent the plane ride strategizing for the conference. We planned our attack for the small-group sessions to make sure we covered every topic we wanted to learn about, and we listed the names of scientists, researchers, and parents we needed to meet. Upon arrival in Cincinnati, we took a taxi to the hotel through the city, which always gives me a shot of adrenaline (ever since I moved to the country). I felt like I was attending a high-powered business meeting.
When we entered the hotel lobby, I quickly crashed. Many families were getting there at the same time, and I was not expecting to see so many children with DMD. There were boys who look just like Charley: darting in and out of the elevator with their siblings, enjoying the excitement of a new place. There were kids who are clearly slowing down, with the tell-tale lordotic stance (curved spine) and irregular gait. And there were boys in wheelchairs, who needed help with everything from opening the doors to cutting their food. Seeing this range of DMD children put it all together for me: my child is very sick.
Although I have devoted my life to finding a cure for Duchenne, a part of me still believes that Charley is okay. Whenever I pick him up from a playdate, I fish around for information from the parents. I always hope they say, "I can't believe he has muscular dystrophy. You would never know it. He seems so active." These words help me believe that Charley is "and will always be" fine. Seeing all those kids at the conference was a punch in the stomach, temporarily knocking me off my feet but ultimately revving me up for action.
So that explains why it's been three weeks since my last blog entry. I have been spending every minute working on the fund, trying to raise money. We have three fundraising events coming up:
Gems for Duchenne, a cocktail party/jewelry auction on Sept. 10 in Lenox, MA
A dinner party at the home of a close family friend.
A David Grover concert for families on May 14 in New York City.
Please check our events page to learn more about these fundraisers.
I always like to end on a positive note, so here's a story. Yesterday I received a call from the printer who does our letterhead.
"We have a problem," she warned.
"What else is new?" I thought.
"You have so many Founding Supporters that they won't all fit on the page," she explained.
Now that's a problem I can deal with.