spinal muscular atrophy

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Novartis axed Avexis execs ahead of Zolgensma scandal

The plot thickens at Novartis, which is becoming embroiled in a scandal over falsification of data related to its rare disease gene therapy Zolgensma.

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US senators blast Novartis for Zolgensma data falsification

US politicians have urged the FDA to come down hard on Novartis for failing to disclose manipulated data used in the approval of its $2.1 million spinal muscular atrophy (SMA) gene therapy Zolge

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Second death in gene therapy trial prompts investigation

A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte

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