Care of the heart in Duchenne is not a simple business. As a community, we realize:
Fortunately, more than 30 representatives from the cardiac care, cardiac research, the National Heart Lung and Blood Institute (NHLBI) and the FDA agreed, and were gracious enough to give two days of their very busy lives to discuss just these issues. These are professionals who deeply care about the Duchenne community and who have worked tirelessly in this field. The passion in the room was palpable, and the vast amount of knowledge was astounding. A formal executive summary will be forthcoming from NHLBI, but I will give a brief summary here.
The meeting was organized by Jonathan Kaltman (NHLBI), Brian Denger (PPMD), and myself (PPMD), and was led by Elizabeth McNally (University of Chicago) and Larry Markham (Vanderbilt University). The following questions were addressed:
Diagnosis and monitoring
Feedback from the FDA indicated that they are interested in receiving recommendations for cardiac surveillance and markers for current and future clinical trials, from this meeting. A letter detailing these recommendations will be immediately forthcoming.
Results from the working group indicated that three major small groups should be formed. These small groups will update and summarize current knowledge, and will make recommendations regarding for future research in each area. The three areas identified include: clinical cardiac care, animal research and human research. Leaders have been proposed and initial reports are expected within six months.
The Duchenne heart is complex to investigate and manage, but not impossible. Sometimes it’s about having the right mix of people in the room, and this was the best of the best.
To learn more about PPMD’s Cardiac Initiative and other cardiac care issues in Duchenne, visit our website.