Novartis has disclosed seven previously unreported cases of tumors in children who received Zolgensma, its gene therapy designed for spinal muscular atrophy. In total, the pharmaceutical company has received nine tumor reports since the treatment gained approval in 2019. Separately, Amgen's drug Repatha has been linked to a reduction in heart attacks.
Key points
Novartis has disclosed seven previously unreported cases of tumors in children who receiv…: Novartis has disclosed seven previously unreported cases of tumors in children who received Zolgensma, its gene therapy designed for spinal muscular atrophy.
In total, the pharmaceutical company has received nine tumor reports since the treatment…: In total, the pharmaceutical company has received nine tumor reports since the treatment gained approval in 2019.
Separately, Amgen's drug Repatha has been linked to a reduction in heart attacks.: Separately, Amgen's drug Repatha has been linked to a reduction in heart attacks.
Sources checked by our analysis pipeline: 1
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.
Pithy intro text about the biopharma milieu.
Novartis cites more tumor reports after Zolgensma
Novartis has disclosed seven previously unreported cases of tumors in children who received Zolgensma, its gene therapy for spinal muscular atrophy. The company has received nine tumor reports in all since the treatment was approved in 2019, STAT’s Ed Silverman and Jason Mast write.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Clinicians and families now face a more complicated decision when considering adeno-associated viruses to deliver therapeutic genes to patients. The recent emergence of cancer cases linked to Zolgensma provides strong evidence that resolves a twenty-year scientific debate over these delivery tools. Medical experts warn that the confirmed risk will shift treatment evaluations, especially for individuals diagnosed with spinal muscular atrophy.
Health officials in the enclave have documented 260 congenital anomalies between January and September of 2026, marking a sharp increase compared to prewar levels. Medical professionals suspect that maternal malnutrition, devastated healthcare services, and environmental pollutants linked to ongoing hostilities are driving the surge. While the exact causes remain unconfirmed, local practitioners are urging an independent international scientific investigation into maternal conditions.
Under a new distribution arrangement, the pharmaceutical giant is leveraging a popular digital health platform to broaden access to its weight-loss treatments. By integrating directly into the telehealth and wellness marketplace, the company can connect patients with its specialized medications through virtual care channels. This strategic collaboration significantly streamlines direct consumer access to targeted obesity management solutions.