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Project summary for the four year $500,000 grant:
The study titled "Modulators of Epigenetic Disease" will use recent advances in understanding the cause of FSHD to develop resources that can be used to identify drugs or other agents that might improve or cure FSHD. Prior work funded by Friends of FSH Research has identified the expression of a gene called DUX4 as the cause of FSHD, and ongoing studies are identifying how the activity of DUX4 might damage the muscle cells. This new project, funded by Friends of FSH Research, will develop cells that can be easily monitored for DUX4 activity and show that these cells can be used to identify drugs that would be candidate treatments for FSHD. This will be an important step toward attracting the interest of pharmaceutical companies and developing a treatment for FSHD that can be advanced into clinical trials.