BTZ has also been tested in several animal models of muscular dystrophy, including dystrophin deficiency (Duchenne, Becker) and laminin α2 deficiency (MDC1A and LGMDR23), with data showing promising therapeutic potential (Araújo et al., 2009; Gazzerro et al., 2010; Carmignac et al., 2011; Körner et al., 2014). This evidence concerns the gene CASC3 and neuromuscular disease caused by qualitative or quantitative defects of dystrophin.