Dr. Subha Raman, Ohio State University, 3 years, $92,000
Start Date: Selected for funding by both the NIH and Cures Within Reach
Duchenne muscular dystrophy (DMD) is a deadly X-linked disease affecting 1 in 3,500 males. DMD patients suffer significant disability due to muscle issues, including to their heart muscle. Current guidelines advocate initiating early heart protective treatment, yet this treatment paradigm has not improved survival much beyond the third decade of life. Potentially promising approaches like gene therapy will take considerable time to improve outcomes.
This research group has recently shown in a DMD mouse model that certain existing drugs typically reserved for advanced heart failure patients can preserve cardiac muscle function at 80% of normal and near-completely prevents fibrosis development. This research group plans to execute a randomized, controlled clinical trial of one of these drugs plus the current standard of care vs. the current standard of care alone in patients with DMD. The treatment group is expected to show significant delays in heart disease using highly reproducible imaging biomarkers selected for efficient sample size design.