In the clinical trial world, there are some things that divide us these days- most specifically, what mutation type your son has. However, we are united in wanting better ways to look for the benefits of potential drugs; who isn’t hoping for a day when the muscle biopsy is a relic from the past?
There are several dedicated research teams doing exciting work to evaluate different ways of measuring changes to muscles. At PPMD, we’ll work hard to keep the community up-to-date about these efforts as results become available. We are also working hard to encourage sponsors of clinical trials to measure changes to muscles by means OTHER than the muscle biopsy, whenever possible.
Your family can help us move closer to a goal of no more muscle biopsies, as well as learning a lot about muscles in Duchenne. The ImagingDMD study is looking for a few more good “men;” specifically, they are looking for males between 5 and 14 with Duchenne. At their first visit, participants must be able to walk roughly the length of a football field by themselves. Boys in the study can be on steroids, or not. The study sites are at the University of Florida, Children's Hospital of Philadelphia, Oregon Health and Science University, Shriners Hospital for Children-Portland, and the University of Pennsylvania. PPMD has provided financial support for this important study.
At our 2012 Connect Conference, we heard from a panel of children and teens about participating in research. One thing uniting these guys was that they were all in the ImagingDMD study. You may remember their positive comments about being in this trial. As the moderator, I was impressed by their enthusiasm for the study and the study teams—the word “fun” was even mentioned more than once!
Please visit the study website at ImagingDMD.org to learn more, or to contact the research teams at each site. The “about” section has a clear explanation of what your son would do if he was in the study.
Holly Peay, Vice President, Education & Outreach
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