As Sarepta fights for Duchenne therapy, another group of patients feels left behind

Sarepta Therapeutics is fighting to salvage its Duchenne therapy Elevidys amid safety setbacks. But families of children with another condition, limb-girdle muscular dystrophy, say they’ve been abandoned by the company, which recently scrapped nearly a decade of work on gene therapies for the disease.
The decision has devastated parents, many of whom uprooted their lives to join Sarepta studies, believing their children were next in line for an effective treatment, STAT’s Jason Mast writes. The company has cited financial reasons for making a hard decision said said it intends to find partners to take on its programs. But advocates now fear years of progress are lost — and worry whether any drugmaker will pick up the torch.

“Families in our community are left asking hard questions right now in rare disease drug development,” Kathryn Bryant, head of the advocacy group Speak Foundation, told STAT.  “Families want their children to have the right to try these treatments.”

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Epic announces an AI overhaul

A giant auditorium is filled with purple light from screens on stage at Epic's annual meeting.

Epic

At Epic’s annual customer meeting in Wisconsin this week, CEO Judy Faulkner announced that the electronic health records system giant will be integrating several new artificial intelligence features into its software. The additions include an AI scribe powered by Microsoft to rival products from health tech unicorns like Abridge, and AI assistants targeted at doctors, patients, and administrative staff.

With more than 40% of the EHR market for hospitals, Epic’s approach to AI reflects its dominance, STAT’s Brittany Trang reports. It kept a relatively low profile while others raced to edge out the competition with announcements for back-office coding tools, physician co-pilots, armies of voice AI agents, and more. Read Brittany’s story on the announcement, and why the company’s slow-walking isn’t the same as sleepwalking.

Automated coding and revenue cycle companies

Revenue cycle management is one of the hottest areas for health tech development. In an example of the scale, Waystar just spent $1.25 billion to acquire Iodine, a company that simplifies claims processing for providers. And there are plenty of other automated coding companies, both AI-powered and not, that have so far been relatively unbothered by Epic’s existing automated RCM tools. But Epic’s new admin assistant Penny introduces new AI revenue cycle tools for autonomous coding, autonomous billing follow-up, reporting about denials, and more. Maybe it can make inroads.

New heart risk model reshapes statins debate

A new risk calculator for atherosclerotic cardiovascular disease, called PREVENT, is redefining who qualifies for statins. But fresh data suggest that lowering the treatment threshold from 7.5% to around 3% over 10 years would keep eligibility roughly the same, STAT’s Liz Cooney writes, while better targeting those most likely to benefit from treatment.

The analysis, published in JAMA Cardiology, highlights both the promise and pitfalls of statins: They cut heart attack and stroke risk by up to 40% but raise diabetes risk in some patients. As the American Heart Association and American College of Cardiology prepare updated guidelines this spring, experts stress the decision is less about numbers alone and more about patient-by-patient tradeoffs — balancing prevention with side effects in the nation’s leading cause of death.

“There are some circumstances for which the answer is obvious: The person who had a heart attack should be on a statin,” a primary care physician not involved in the study told STAT. “At a certain point, what is a benefit that’s real but so small it’s not worth it? It’s a combination of complex science, but also subjective human values.”

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