No­var­tis' star gene ther­a­py faces po­ten­tial safe­ty is­sue fol­low­ing a re­cent in­fant death in study

No­var­tis got some more up­beat press ear­li­er in the week when re­searchers on the Zol­gens­ma gene ther­a­py pro­gram out­lined some up­dat­ed bio­mark­er da­ta on their SMA drug and not­ed that 21 of 22 pa­tients were alive and event free as of last fall. But it turns out there was a post script to the study that rais­es some safe­ty con­cerns for the phar­ma gi­ant’s ther­a­py — re­cent­ly ac­quired in an $8.7 bil­lion ac­qui­si­tion of AveX­is.

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