In May 2019, the FDA had approved Zolgensma, the first gene therapy to treat children less than two years of age with spinal muscular atrophy (SMA), and bi-allelic mutations in the SMN1 gene, the most severe form of SMA and a leading genetic cause of infant mortality. Zolgensma, the world’s most expensive treatment (>$2 million), is currently being reviewed under Accelerated Assessment Procedure in the EU.