DYSF and neuromuscular disease caused by qualitative or quantitative defects of dysferlin: We developed a therapeutic pipeline to identify small molecules capable of restoring function to loss-of-function DYSF patient missense mutations (DYSFPMMs), which represent 30%–40% of dysferlinopathy mutations (Cacciottolo et al., 2011; Jin et al., 2016; Schoewel et al., 2012).