AGRN and neuromuscular disease: Our findings point to an unforeseen therapeutic approach, as this strategy does not directly target the mutant protein but rather targets a wild-type protein that has diminished activity caused by the mutation of an upstream gene, in this case DOK7. Epistatic rescue in this way could also provide therapy for CM caused by mutations in AGRIN, LRP4 or MUSK, in addition to DOK7, as well as for other neuromuscular diseases.