Skip to Main Content

And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is still being discussed, but we hope to hang a bit with our short person, catch up on our reading, and have a listening party with Mrs. Pharmalot. And what about you? The time for a summer getaway is nigh, so you can whip up something fun. You could also hit the pause button and catch up on some reading or station yourself in front of the telly. Or you could use this as an opportunity to touch base with someone special and take stock of what matters. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon. …

Two children have died from acute liver failure after being administered Zolgensma, a pricey gene therapy sold by Novartis to treat a rare disease, STAT reports. The company this week began notifying physicians and payers about the deaths, which occurred over the past few months in Russia and Kazakhstan. Although acute liver failure is noted in the product labeling information, a Novartis spokesperson acknowledged these episodes marked the first fatal cases attributed to the side effect. There were no deaths tied to acute liver failure in clinical trials for the medicine, which is used to treat spinal muscular atrophy, a genetic disorder that causes muscle wasting and weakness.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED

Create a display name to comment

This name will appear with your comment