RT
August 12, 2022
Two children died of acute liver failure after being treated with Zolgensma gene therapy, which is often referred to as the most expensive drug in the world, the medication’s manufacturer Novartis said on Thursday.
Acute liver failure is a “known adverse event” of the drug, which is used in patients suffering from spinal muscular atrophy (SMA), the Swiss pharmaceutical company said, but these are the first fatal cases caused by this complication. The patients had received corticosteroids, Novartis said, to prevent severe liver damage.