Of note, preliminary results, as of the 11 June 2021, of phase 3 SPR1NT clinical trial (NCT03505099) demonstrated significant therapeutic benefits of onasemnogene abeparvovec in pre-symptomatic SMA patients aged ≤6 weeks with 2 or 3 copies of SMN2, with some patients achieving gross motor milestones similar to non-SMA infants [22, 23]. This evidence concerns the gene SMN2 and proximal spinal muscular atrophy.