Given the extremely low rate of FSHD-associated inappropriate expression of DUX4 at the myoblast, myotube, and muscle stages, many of the FSHD-dysregulated transcription-regulatory or cell signaling genes revealed by our expression profiling may be more effective targets for developing pharmacologically-based or gene therapy-based treatment of FSHD than DUX4 itself. Here, DUX4 is linked to facioscapulohumeral muscular dystrophy.