Although there is much hope for this drug, it has been known to present some complications since its pre-clinical testing. For example, nusinersen is not incorporated efficiently into certain cell types and tissues8. Repeated intrathecal injection is employed for its administration in the clinical trials14, because it cannot easily cross the blood-brain barrier8. Although intrathecal injections of nusinersen are considered to be safe, the typical side effects associated with lumbar puncture are induced in nearly one-third of treated patients in the clinical trial15. In addition, the manufacturer has announced that the treatment will cost $750,000 in the first year, and $375,000 every following year16. Development of a new and more affordable alternative would bring practical benefits for the patients.