“1. Funding of Dr. Michael Kyba’s project at the University of Minnesota to conduct a massive screening experiment looking for inhibitors of the protein DUX4 that causes FSHD. Finding a mechanism to inhibit DUX4 from doing damage to muscle is a critical step towards developing treatments. Using a platform developed by Dr. Ali Tavassoli at the University of Southampton, Dr. Kyba is able to test tens of millions of potential small molecules for their ability to inhibit DUX4. Dr. Kyba has recently published his first results from the first DUX4 inhibitor screening effort.”

“2. Funding for a proposal by Dr. Daniel Miller and Dr. Rabi Tawil at the University of Washington and University of Rochester. Dr. Miller and Dr. Tawil have collected serum samples from a group of patients who volunteered to donate blood samples to the laboratories. They will test the blood using a novel technique to search for serum biomarkers that are specific for FSH Muscular Dystrophy. Dr. Miller and Dr. Tawil hope to identify new and robust markers that can be used in later stage clinical trials, but by testing a wide array of serum biomarkers, critical insights into the mechanisms of muscle damage may be found.”