biotech
After a child's shocking death, what's next for gene therapy?

Maria Fabrizio for STAT
Since 2020, more than $800 million has been invested into companies working on gene therapy treatments for at least a dozen different brain disorders. But money can't solve every problem, and at least a dozen patients have died in relation to the treatments. This summer, a young child was the first person to receive a new experimental gene therapy, engineered to overcome previous safety and efficacy challenges. Just days later, the child died.
"This is an outlier that, to me, is the most material event that I've seen in the field of genetic medicine for 10 years, where there was no suggestion that this was going to happen," said Jim Wilson, a pioneer in the field. "And this is scary, I'm sure, for all those involved."
Read more from STAT's Jason Mast about how the death has sent concern and uncertainty rippling through labs and companies developing gene therapies, and what changes advocates and academics are calling for to prevent — or at least better protect against — future tragedies.
one big number
£13 million
That's how much it costs the U.K.'s National Health Service each year to treat women with sudden heavy menstrual bleeding. (Most people think of heavy flows as a chronic issue, but it's possible to experience in an acute episode, where a blood transfusion may be needed.) This cost estimate comes from a study published last week in The Lancet Obstetrics, Gynaecology, & Women's Health, which assessed data on more than 1,000 women who were admitted to the hospital with acute cases of heavy menstrual bleeding in the first half of 2024.
The findings signal an "urgent need" for better clinical strategies and early interventions to manage heavy menstrual bleeding, especially in these cases of sudden, strong onset, the authors write.
quote of the day
'The ventilator wasn't keeping Michael alive. It was keeping the rest of us from having to say goodbye.'
That's from a First Opinion essay published today by palliative medicine physician Raya Elfadel Kheirbek. In the piece, she recounts an experience being called in to help a family understand that even though their loved one didn't look dead, a fatal injury in his brain meant that he wouldn't ever come back.
"The science of medicine told us Michael was gone," Kheirbek writes. "But the art of medicine, and the work of palliative care, meant holding space for his family's grief, hope, and faith." Read more.
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