I have had the great pleasure of representing Parent Project Muscular Dystrophy and the Duchenne community at the Muscular Dystrophy Coordinating Committee (MDCC). Members of the MDCC are involved in muscular dystrophy research, including federal agencies and advocacy organizations. Last August, I spoke at a MDCC meeting. The meeting was seven months after my son Matthew died from a cardiac arrhythmia. His passing made me realize the management of cardiac health is complex and needs to be better understood.
In my presentation, I talked about the need for an increased understanding of heart health for Duchenne making three points. I spoke about a limited standard of care for the heart, the potential impact of new drugs and therapies on the heart, and the possible role of approved medications for people with Duchenne. I directed my comments to MDCC members from the National Heart Lung and Blood Institute. I asked them to consider working with PPMD to better understand cardiac health.
At the end of my presentation, Dr. Gail Pearson from NHLBI asked if she and her colleague Dr. Jon Kaltman could contact me to talk about my ideas. A call was organized that included Pat Furlong to discuss an NHLBI and PPMD collaboration for a working group focusing on cardiac issues related to Duchenne. The development of the meeting is underway to be led by Dr. Elizabeth McNally and Dr. Larry Markham. We’ll all benefit from this collaboration.
Families affected by Duchenne muscular dystrophy do not have the luxury to waste time. No one therapy will do everything to fully help those living with the disorder. Looking at as many options as possible and focusing on the most promising is crucial for all families. The NHLBI and PPMD Cardiac Working Group is part of the process. I am optimistic these efforts will be useful in understanding cardiac issues.