Novartis has recorded two deaths after treatment with its spinal muscular atrophy gene therapy Zolgensma, once again bringing gene therapy’s safety into attention.
Two children in Russia and Kazakhstan died about five to six weeks after receiving Zolgensma, Novartis confirmed to Fierce Pharma. Both patients died of acute liver failure, a known side effect of Zolgensma that’s included in a boxed warning on the one-time therapy’s label. Both had received corticosteroid taper to restore liver function…
