Sarah Jenssen is the rarest of the rare — a girl with Duchenne muscular dystrophy, which almost always afflicts boys, as STAT wrote about in 2019. She wasn’t eligible to enroll in clinical trials for cutting-edge Duchenne therapies, but her family was elated when the FDA this year approved a gene therapy called Elevidys from Sarepta Therapeutics for nearly all patients, and didn’t limit it to boys.

As STAT wrote in September, Sarah’s insurance initially denied her the treatment, saying it was considered experimental for patients whose conditions had progressed to the point that they depended on wheelchairs — patients like Sarah, now 15. 

After the family appealed, however, the insurer reversed the decision. Earlier this week, Sarah received a dose of Elevidys from her specialists at Vanderbilt. Sarah’s mom, Deb, told STAT she knew of one other girl who had been treated so far. 

The debate continues over the quality of evidence underlying Elevidys’ approval, which patients should get it, and whether the FDA erred in approving it at all. But for the Jenssens, the hope is that the gene therapy can halt or at least delay any further progression of Sarah’s disease and give her a longer life. As Deb told STAT in September, “Even though she’s in a wheelchair, she can eat, she can use the bathroom by herself, she can get herself dressed in the morning. She’s still a pretty independent person. But I think we’re months away from losing all that.” — Andrew Joseph

Public Health

U.S. life expectancy to dip below other high- and some middle-income countries

The Institute for Health Metrics and Evaluation at the University of Washington published new life expectancy projections in The Lancet yesterday, and the outlook for Americans is not good: Between 2022 to 2050, Americans’ life expectancies will only increase from 78.3 to 80.4 years. The modest increase will drop the U.S. life expectancy ranking from 49th to 66th of 204 countries.

The biggest risk factors are familiar to all of us: high BMI, high blood pressure, diets high in sodium and processed meat, and alcohol intake. The researchers forecasted that reducing smoking and drug use would also improve mortality dramatically — by 2050, they calculated, the U.S. will have the highest drug-related mortality in the world, more than twice as high as the second-ranked country, Canada.

REGULATIONThe thorny path ahead on lab-developed testsA few months ago the FDA finally made plans to regulate lab-developed tests, or LDTs, in order to improve test safety and reliability. But that’s sparked controversy, and labs have launched lawsuits to challenge the agency’s authority. Meanwhile, patient groups and traditional diagnostics companies support regulatory oversight in order to prevent harm from unvetted tests.But the FDA’s plans to shift policy could very easily be derailed — particularly with Donald Trump reentering the White House next month, STAT’s Lizzy Lawrence writes.“I would say there’s a lot more caution in the community to wait and see what happens with litigation, the new administration, and the final rule,” one diagnostics CMO told STAT.Read more.