Crazy how this broke... first came out via patient advocates on social media and then parents of children in the trial talking via facebook groups. Parents relayed what their neurologists told them and there are claims that Novartis was withheld and rolled out safety information in tiers.

Sounds like there are multiple spinal tumors and at least one case of an adrenal tumor. AAV gene therapy is promising but still has a lot to be discovered regarding risk.

Interestingly Novatis already got dings for manipulating Zolgensma preclinical data: https://www.pharmamanufacturing.com/compliance/regulatory-gu...

> On Aug. 6, the U.S. Food and Drug Administration (FDA) publicly rebuked the Swiss pharmaceutical company Novartis for manipulating data involving its $2.1 million gene therapy. (1) The FDA said that Novartis’ gene unit AveXis failed to report the falsified data prior to its gene therapy treatment Zolgensma getting agency approval in May 2019 despite the fact that the company knew of the issues as early as March 2019.