Two groups, Marsollier et al. and Chen et al. reported the efficacy of antisense phosphorodiamidate morpholino oligonucleotide to suppress the expression of DUX4 in both a FSHD cell model [71,72] and FSHD patient muscle xenografts in mice [72] by targeting the polyadenylation signal of DUX4. They also demonstrated that the PMO can decrease the abnormally high expression of many genes downstream of DUX4 pathway. Here, DUX4 is linked to facioscapulohumeral muscular dystrophy.