Even if the mouse in which TAs are injected with pCIneo-DUX4 by hIMEP does not recapitulate all the complexity of FSHD pathophysiology, this model has an added value in the forefront of pre-clinical evaluations, particularly in a context in which high throughput therapeutic screening is still necessary. Here, DUX4 is linked to facioscapulohumeral muscular dystrophy.