Using the same FSHD-like transgenic mouse model, but without inducing DUX4 expression, Bouwman and colleagues have since reported that the longitudinal delivery of a constrained ethyl gapmer antisense oligonucleotide targeting the open reading frame of DUX4 is also effective at knocking down the leaky DUX4 level and alleviating muscle pathology, although with limited improvement in muscle mass and function [21]. The gene discussed is DUX4; the disease is facioscapulohumeral muscular dystrophy.