A child in Russia died after receiving Zolgensma, the gene therapy used to treat spinal muscular atrophy (SMA). Reports indicate the cause was acute liver failure that developed between five and six weeks after the infusion. Zolgensma was registered in Russia at the end of 2021, and official statements confirm this timeline and the patient’s condition. The information comes from coverage by Reuters and details provided by the drug’s manufacturer, the Swiss company Novartis.
In addition, a child from Kazakhstan who had SMA died with a similar presentation. The reporting on these cases underscores ongoing concerns about safety signals associated with this therapy in real-world settings, and it highlights the critical importance of monitoring patients closely after infusion.
Earlier statements from the Health Ministry indicated expectations that a Russian SMA treatment could enter the market by 2026, outlining a plan to broaden access within the country. This framing reflects ongoing policy discussions around pricing, reimbursement, and the availability of advanced therapies for SMA in Russia and neighboring regions, as reported by health authorities and major news outlets.
SMA therapies, including Zolgensma, are among the most expensive and technically complex medicines in modern healthcare. They aim to modify the disease process with a single treatment, potentially altering the long-term health trajectory for affected individuals. While Zolgensma offers the promise of effectively halting SMA symptoms when administered early, the optimal approach remains early detection and prompt treatment, ideally before age two. The price for a single Zolgensma dose has been cited at approximately $2 million, a figure that has sparked ongoing discussions about affordability, access, and value in healthcare systems across North America and beyond.