Zolgensma is indicated for the treatment of:• Patients with 5q spinal muscular atrophy (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 3 copies of the SMN2 gene. The gene discussed is SMN1; the disease is spinal muscular atrophy.