FDA probes Zolgensma gene therapy for cancer risk in children

The FDA is investigating whether Novartis' gene therapy Zolgensma, priced at over $2 million, may cause malignant tumors in treated children. The investigation follows emerging safety signals, though no causal link is confirmed. This matters because Zolgensma is a one-time curative treatment for spinal muscular atrophy, and any cancer risk would have major implications for gene therapy regulation and patient safety.

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