Specifically, we hypothesized that the DUX4 transcript may be naturally regulated by one or more of the 1917 known endogenous human microRNA genes, and that it may be possible to upregulate a DUX4-targeting microRNA with a small molecule, thereby offering a potential drug-based RNAi treatment for FSHD. The gene discussed is DUX4; the disease is facioscapulohumeral muscular dystrophy.