Gene therapy successes point to better therapies

The drug Zolgensma was recently in the news for all the wrong reasons. In August, the US Food and Drug Administration (FDA) gave drug manufacturer AveXis, Inc, a subsidiary of Novartis AG, a major slap on the wrist for violations related to the approval of Zolgensma, a new treatment for spinal muscular atrophy (SMA). The agency said the company had failed to promptly report to the proper regulatory authorities issues of datamanipulation in some product testing.Ominous newspaper headlines followed…

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Fonte: 23866–23870 | PNAS | November 26, 2019 | vol. 116 | no. 48 www.pnas.org/cgi/doi/10.1073/pnas.1918306116