PPMD is excited to award Dr. Jonathan Soslow (Vanderbilt University Medical Center), and his collaborators Drs. Larry Markham (Riley Children’s Hospital) and Christopher Spurney (Children’s National Medical Center), a $65,000 grant to characterize microRNA and serum protein cardiac biomarkers to predict heart disease in Duchenne. This work helps to advance our understanding of Duchenne cardiomyopathy and develop predictive cardiac biomarkers.

This grant was made possible, in part, thanks to your contribution to PPMD’s Cardiac Initiative.

Dr. Soslow explains the importance of this work:

“Heart disease is the leading cause of death in individuals with Duchenne and Becker muscular dystrophy.  To develop novel therapies for heart disease in Duchenne, we must be able to better identify patients with severe disease and predict who will develop rapid progression.”

Dr. Soslow has helped create an FDA funded consortium of eight of the largest institutions with Duchenne cardiomyopathy expertise, the DMD Cardiovascular Care Consortium (DMDCCC), to identify DMD surrogate outcome measures. Preliminary data from this consortium has identified potential microRNA and protein serum biomarkers of cardiomyopathy in DMD.

This work leverages the DMDCCC’s work with an ongoing natural history study to evaluate novel blood biomarkers with significant potential to function as predictors of cardiovascular disease progression. The study will build on Dr. Spurney’s preliminary data to generate a panel of microRNA biomarkers that could be used to assess cardiovascular disease severity and predict future cardiac disease progression.

This study will also validate separate serum protein markers that characterize fibrosis and may also aid in predicting future changes in left ventricular ejection fraction (LVEF). The results of this study will facilitate comprehensive testing of future targeted therapies, which is crucial for lengthening and improving the quality of life of individuals with Duchenne.

Dr. Soslow Discusses Duchenne and Becker Cardiac Care
at PPMD’s 2023 Annual Conference