To date, Zolgensma® is indicated for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene [9]. The gene discussed is SMN1; the disease is spinal muscular atrophy, type 1.