Another candidate experimental therapy – overexpression of the LARGE enzyme – has been shown to restore αDG function in a variety of α-dystroglycanopathies caused by mutations in FKRP, FKTN, POMGNT1 and POMT1 (Barresi et al., 2004; Yu et al., 2013; Vannoy et al., 2014). This evidence concerns the gene LARGE1 and neuromuscular disease caused by qualitative or quantitative defects of alpha-dystroglycan.