Novartis said it has received nine reports of tumors in children previously treated with its spinal muscular atrophy gene therapy Zolgensma, with two cases showing evidence consistent with a possible link. Patient advocacy groups raised concerns after new disclosures to families and clinicians, and Novartis noted that seven additional reports have not been causally connected to the therapy. The updates revive safety discussions around AAV vector-related tumorigenicity signals and highlight the monitoring expectations for gene therapies years after dosing. Regulatory review is underway for the new reports, according to the account. For the gene-therapy market, the development adds near-term uncertainty to long-term risk communication and may affect how clinicians and families weigh benefits versus emerging signals.
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