“…Clinical management involving physiotherapies, vision and hearing aids, orthopedic interventions, pain and fatigue management or surgical scapular fixation has shown some clinical benefit and improved the quality of life for FSHD patients ( 49 , 50 ). Since aberrant expression of the DUX4 gene has been extensively reported as the main causative factor of FSHD ( 9–14 , 20 ), pre-clinical strategies silencing DUX4 expression have shown promise for FSHD treatment ( 20 , 22 , 24 , 29 , 41 , 51–53 ). Among antisense approaches, those targeting the pLAM region of DUX4 3′UTR, including ours, have provided the best down-regulatory effect ( 20 , 24 , 29 , 30 ).…”