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Lucy may not live to see her second birthday without expensive gene therapy

Lucy Van Doormaal was born on April 1 in Vancouver, but before her parents, Scott and Laura Van Doormaal, could ...
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Unconventional drug ‘lottery’ will hand out 50 doses of Zolgensma, world’s most expensive gene therapy

The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It ...
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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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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 ...
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Viewpoint: Outrage over $2 million price tag for new gene therapy is perplexing and disappointing

As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and ...
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$2.1 million gene therapy is most expensive drug in history

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the ...