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Viewpoint: Spinal muscular atrophy treatment Zolgensma a gene therapy trailblazer, despite controversy

In August, the US Food and Drug Administration (FDA) gave drug manufacturer AveXis, Inc, a subsidiary of Novartis AG, a ...
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Animal safety concerns prompt FDA to halt study testing Zolgensma gene therapy for spinal muscular atrophy

The Food and Drug Administration has halted a clinical trial of Novartis’ Zolgensma gene therapy due to a safety concern ...
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Application for pricey gene therapy Zolgensma contained manipulated data, but drug will remain on market, FDA says

The Food and Drug Administration said Tuesday [August 7]  that Novartis submitted manipulated data as part of its application for ...
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Growing criticism over stratospheric cost of spinal muscular atrophy-fighting drug Zolgensma ignores trade-off value

Novartis recently won FDA approval for Zolgensma, a new medicine that can potentially cure infants suffering from the otherwise fatal ...