DUX4 and facioscapulohumeral muscular dystrophy: Several ASOs have shown beneficial results in patients with neuromuscular disorders.29, 30, 31 For FSHD, no ASOs have yet been tested in patients; different studies, however, have shown that ASOs were efficient in reducing DUX4 and DUX4 target genes in FSHD myocytes and in FSHD mice.15, 16, 17, 18, 19,21 A major advantage of our systemic approach compared to most other in vivo studies in FSHD mice is that all tested skeletal muscles were targeted by the ASO instead of one muscle or a part of the muscle.