There is undoubtedly greater hope than ever before in the world of DMD. More funding is going into research than every before, and yet, with all of the research that is going on, clinical trials have yet to demonstrate convincing efficacy to the necessary regulatory agencies. Unfortunately, the plural of anecdote is not data, but stories, like that of Billy Ellsworth, suggest that the drugs work, and a cure is right in front of us.
Today, Biomarin, the producer of drisapersen, is going before the FDA to seek approval of their drug to be distributed to individuals with the appropriate genetic mutation.
Just recently, the Washington post eloquently highlighted the hope that our DMD family shares as we continue to fight this disease. The article (and associated video) are certainly worth a quick glance.
A few years ago, I was asked by one of my best friends, Lt. Nicholas Perry, MD, if I'd like to run a marathon with him. He was going to be visiting his significant other's family in Orlando right around the time that the Disney Marathon took place. Being one to not back down from challenges / letting my stubborn pride get the best of me, I agreed to the challenge. Prior to this, I had run a few 5k's, but nothing more than a few miles at a time. With a bit of support from friends and family, a dedicated training plan, and strict diet, I was able to finish the full 26.2 miles.
I didn't know if I'd continue running or not after the marathon (and in full disclosure, haven't done another marathon since). I haven't been able to escape running though, be it through a short few miles at a time, or half marathons, I still find myself going back to running whenever I get the chance.
Why do I run? I ask myself that all of the time. There's no single reason for why I run, but here's a few thoughts that spring to mind:
If there's anything this PhD (and entirety of school) has taught me, it's that life is a journey, and just like going for a long jog, the experiences along the way are more worthwhile than crossing the finish line.
Recently, the other grad students in the lab went to the Myology Course at Nationwide Children's Hospital in Columbus, Ohio, leaving me as the only student remaining in the lab. I tried to make the most of it by decorating our office with bubble wrap, police tape, and a number of small oddities, personalized for each student. The capstone was rigging an airhorn to several friends' chairs, as you can see below in the videos.
It's important to at least try to have a little bit of fun during this grind of a PhD!
Just a few weeks ago (sorry for the delayed post), we held our annual autumn fall retreat. The purpose of this retreat is severalfold: A) to introduce the new scholars to our program and everyone else, B) learn about the breadth of research that our scholars are investigating, and C) build a sense of camaraderie and family amongst our program, from faculty to students. We held the retreat at the President's House (at my suggestion, if I may), on a lovely blue skied fall Saturday (of course, during a weekend that UF was not playing football). From the most basic benchtop discoveries to investigations on drug reform in Malaysia, our scholars covered a diverse range of topics during the day.
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies