Stimulation of the agrin-Lrp4-MuSK-Dok7 pathway, e.g. by Dok7 gene therapy or an engineered agrin protein, has a therapeutic effect in animal models for AChR MG12, congenital myasthenic syndrome (CMS)13, ALS14, SMA15,16 and several forms of muscular dystrophies17. This evidence concerns the gene AGRN and congenital myasthenic syndrome.