What’s available: 3,000 FDA-approved drugs, countless nutriceuticals and 1000s of devices The Problem: 90% of all patients will not see an effective treatment in their lifetime and for 12,000 diseases, only 30-40 new drugs are approved annually What can be done: With 25 research partners, and less than $7M in philanthropic funds, we have created 9 repurposed treatments changing the lives of thousands of patients What can be done: With 25 research partners, and less than $7M in philanthropic funds, we have created 9 repurposed treatments changing the lives of thousands of patients

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.

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