Exon-skipping therapy is an approved treatment for Duchenne muscular dystrophy [31] and recently, we developed a therapeutic strategy using branchpoint-targeted antisense oligonucleotides for patients with Fukuyama congenital muscular dystrophy caused by an FKTN transcript involving a pseudoexon [32]. Here, FKTN is linked to congenital muscular dystrophy due to LMNA mutation.