PTM molecules designed to replace exons 1 to 5 of the mutated Lmna pre-mRNA, allowing for the targeting of 51% of the described LMNA mutations, were tested in vitro and in vivo in the LmnaΔK32/ΔK32 mouse model, a KI mouse reproducing a LMNA mutation found in severe EDMD and L-CMD [54]. Here, LMNA is linked to congenital muscular dystrophy.