Health authorities examine the link between a gene therapy for a rare pediatric disease and cancer risk

Novartis reported that nine children developed tumors after receiving its gene therapy Zolgensma, with two cases confirmed linked to the treatment. The therapy is for spinal muscular atrophy (SMA), a severe condition affecting one in 7,000. Zolgensma aims to correct the SMN1 gene defect, potentially allowing treated children to live normally. In France, no adverse cases have been reported among the 229 patients treated.