The gene therapy Zolgensma will remain on the market, but Novartis and subsidiary AveXis Inc. could face civil or criminal penalties after revelations by the US Food and Drug Administration that data used for approval had been manipulated and Novartis knew about the problem months before and did not disclose it, probably fearing delays or worse. How could this have happened, why does the FDA maintain confidence in Zolgensma’s efficacy and safety, and will the Agency do things differently moving forward as a result?