News
Gene Therapy Under Scrutiny After Nine Tumor Reports
The Brief
- Novartis acknowledged nine reports of tumors in children treated with Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy.
- The source reports that two of the tumor cases were in the news, but the available text is cut off, so further details are not confirmed here.
Why it matters
Reports of tumors after a gene therapy approved for a serious childhood disease raise questions about the long-term safety of such treatments, which clinicians and researchers need to monitor.
Who’s affected
- Children treated with Zolgensma for spinal muscular atrophy
- Clinicians and families weighing gene therapy for spinal muscular atrophy
- #gene therapy
- #drug safety
- #spinal muscular atrophy
- #zolgensma
- #novartis
- #tumors
(MedPage Today) -- Drugmaker Novartis acknowledged Thursday that there have been nine reports of tumors in children treated with its gene therapy onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy (SMA). Two of the tumor cases were...
Timeline
Novartis acknowledged nine tumor reports in children treated with Zolgensma (reported by MedPage Today)
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Gene Therapy Under Scrutiny After Nine Tumor Reports