Zolgensma is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age:

• With 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or
• With 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene