Our study has shown that the LNA/DNA mixmers we developed have very high efficacy in vitro. Administration of mixmers could have a significant effect in vivo, even with lower doses. From a therapeutic point of view, administration of lower concentrations and minimizing administration frequency would be desirable because of safety concerns and the cost of the treatment. Therefore, LNA/DNA mixmers are one of the promising drug candidates suitable to develop antisense oligonucleotide therapies to treat SMA.