closer look
Hemophilia gene therapies have few takers
Credit uc/lc
You could say Noah Frederick, 23, has been waiting all his life for a gene therapy to cure his hemophilia A. Now two gene therapies are here, but he's not sure he wants one for his bleeding disorder. "I'm on the fence," he said. He's not alone: Only a handful of patients with hemophilia B, the rarer form of the disease, appear to have been treated worldwide since Hemgenix was approved in November 2022. Since Roctavian was approved for hemophilia A last June, only three patients have been treated.
History may hold the explanation: Hemophilia gene therapy was originally conceived during the AIDS crisis, when contaminated blood-clotting products killed thousands of patients. Since then, new treatments, including synthetic clotting factors, radically changed the standard of care. That allows patients to be cautious about a treatment that some data suggest is more of a reprieve than a cure. STAT's Jason Mast explores the question.
medical devices
Experimental patch translates throat movement into speech to help those with voice disorders
In a small, preliminary trial, researchers have tested a soft adhesive patch they say can turn throat movements into speech. Created from a material that converts motion into electricity, the sticky patch changes muscle movements in a person's throat into electrical signals that power the device and flow into a machine-learning algorithm trained to match these moves to words, which are then projected into a speaker.
The study, which appears in Nature Communications, has some big caveats. It was small, testing the patch in only eight people, none of whom have voice disorders. And some people with voice disorders have had their voice boxes removed because of cancer. The research team also needs to add more phrases than the five they tested to the machine-learning algorithm and make the voice customizable. "Consider this a proof of concept demonstration," study co-author Jun Chen said. STAT's Lizzy Lawrence has more.
health
11% of high school seniors may be using Delta 8 THC
Over the last year, 11% of high school seniors said they'd used Delta 8 THC, a new JAMA study based on a national survey reports. That's a lot, Nora Volkow, director of NIH's National Institute on Drug Abuse, said about the quasi-legal psychoactive version of cannabis. "That's at least one or two students in every average-sized high school class who may be using Delta-8."
The popularity of these products, available in edibles resembling snack foods like Flamin' Hot Cheetos, Gummy Worms, and Pop Rocks, has soared after a 2018 legal loophole allowed their sale online, at gas stations, smoke shops, and in many states where traditional marijuana is illegal. While 11% of the 2,186 respondents surveyed reported using Delta 8 in the last year, 68% of users reported using it more than three times, and 17% of users reported using the product more than 40 times. STAT's Nicholas Florko has more.
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