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STAT+: Pharmalittle: We’re reading about a Novartis gene therapy, faster U.K. drug reviews, and more

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Based on a feed excerpt; full article content has not been acquired. Source evidence retrieved 06/11/2026, 14:51:24 UTC.

Briefings

Novartis acknowledged seven previously unknown instances of tumors in children treated with its spinal muscular atrophy therapy, a finding that has raised safety concerns among families, physicians, and advocacy groups. The cases were publicized by an advocacy group and a Facebook page after Novartis disclosed the information to neurologists. Four of the eight people appointed by U.S. Health and Human Services Secretary Robert F. Kennedy Jr. to an influential health panel own stock in companies affected by the group's decisions, with the U.S. Preventive Services Task Force examining the cases. The evidence indicates regulatory authorities are now examining the cases, but the specific outcomes of the investigation remain uncertain.

Key points

• Novartis acknowledged seven previously unknown instances of tumors in children treated with its spinal muscular atrophy therapy.
• Four of the eight people appointed by U.S. Health and Human Services Secretary Robert F. Kennedy Jr. to an influential health panel own stock in companies affected by the group's decisions.

Context

STAT+ reports on a Novartis gene therapy safety issue involving children with spinal muscular atrophy, noting that four of eight panel members hold stock in affected companies. The U.S. Preventive Services Task Force is examining the cases, and regulatory authorities are reviewing the findings. This discovery has raised concerns among families, physicians, and patient advocacy groups regarding the safety of the therapy.

Limitations

The evidence indicates regulatory authorities are now examining the cases, but the specific outcomes of the investigation remain uncertain. The cases were publicized on Thursday by an advocacy group and on a Facebook page after Novartis disclosed the information to neurologists, and the timeline of the initial discovery is not fully detailed.

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