Therefore, the preventive effect of MT-DUX4-ASO in TMX-treated DUX4-TG mice indicates its potential to prevent the emergence of active muscle lesions and, thus, symptomatic disease progression, including mobility impairment in FSHD patients when these patients are regularly treated with MT-DUX4-ASO. Here, DUX4 is linked to facioscapulohumeral muscular dystrophy.