A single treatment that could replace a lifetime of pills. Drugs with this potential have started to receive the stamp of approval from the FDA and hit shelves, offering patients with both common and rare diseases new hope.
But what happens if it stops working several years down the road, or if safety issues emerge? Or, as was recently the case with drug giant Novartis' (NYSE: NVS) new gene therapy Zolgensma, that there were inaccuracies in some of the data?
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