In a statement, the drug company Novartis, which manufactures the drug, confirmed the two deaths. Zolgensma works as a kind of gene therapy and can help children with the muscle disease Spinal Muscular Atrophy (SMA). The drug costs about 1.9 million euros per treatment, according to the company.
Belgium has been paying it back since 1 December 2021. The expensive medicine became known in our country a few years ago when the parents of baby Pia organized a collection to buy it for their daughter.
The two children in Russia and Kazakhstan died of acute liver failure about six weeks after being treated with Zolgensma. Liver failure is a known side effect of the drug. To avoid that complication, patients are given steroids. The children died one to ten days after that administration.
According to Novartis, they are the first patients to die of liver failure after gene therapy. The fatal outcome will now be on the label of the drug and the health authorities have been informed. The pharmaceutical giant also states that – benefits weighed against risks – it remains strongly convinced of the effect of Zolgensma.
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