Two deaths of children reported from Zolgensma treatment for spinal muscular atrophy.- Novartis

The patients developed acute liver failure between five and six weeks after infusion with the gene therapy, Zolgensma and approved to treat spinal muscular atrophy, a rare, inherited condition that in its most severe form is often fatal by age two. While acute liver injury is a known risk of treatment with Zolgensma, these are the first cases that led to patients’ deaths.