Novartis Pharmaceuticals is reporting that two children have died after taking Zolgensma, a gene therapy drug designed to treat a rare condition called spinal muscular atrophy.
In an email statement, Novartis says the children died from acute liver failure about six weeks after receiving an infusion of Zolgensma, and one to 10 days after tapered doses of a steroid meant to combat liver complications.
The deaths occurred in Russia and Kazakhstan.
The FDA approved Zolgensma in the U.S. in 2019 and is estimated to cost more than $2 million per patient.
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