Currently, there are only three FDA and/ or the European Medicines Agency (EMA) approved Phase I or I/II clinical trials aimed at assessing the safety, biological activity, and efficacy in MDs, all of them delivering a shorter version of dystrophin (micro‐ or mini‐dystrophin) in a small cohort of DMD boys (Duan, 2018; Verhaart & Aartsma‐Rus, 2019) and one approved treatment for spinal muscular atrophy (SMA), a neuromuscular disease (Shahryari et al., 2019). The gene discussed is DMD; the disease is spinal muscular atrophy.