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."

cancer

FDA decision on Replimmune has ripple effects

The Food and Drug Administration's surprise rejection of Replimune Group's oncolytic virus therapy for melanoma has already claimed collateral damage, STAT's Adam Feuerstein writes. Krystal Biotech suspended enrollment in a Phase 2 trial of its similar drug, KB707, citing "heightened uncertainty" around accelerated approval pathways.

Both drugs are engineered herpes viruses injected directly into skin cancer lesions. The Replimune decision by regulators "has signaled a shift in their approach to drug approvals," according to a bulletin sent to physicians participating in Krystal's clinical trial.

psychedelics

Opinion: Why the VA shouldn't stall on psychedelics

The Department of Veterans Affairs is embracing psychedelic research, but unless Congress sustains momentum, there's a risk that veterans won't benefit, Reps. Lou Correa and Jack Bergman, along with Marine Corps veteran Juliana Mercer, opine for STAT.

MDMA and psilocybin have shown powerful results in clinical trials, and lawmakers on both sides of the aisle are already pushing the VA to prepare its system, train clinicians, and build infrastructure ahead of FDA approval.

Veterans themselves, who face suicide rates nearly 60% higher than civilians, have been the most credible advocates for the benefits of psychedelic therapy, the authors write. They should not have to leave the country to access treatments that science and bipartisan consensus increasingly support.

"This is not about chasing hype," they say. "Congress must ensure the VA remains resourced and ready to lead — and that we don't allow this moment of progress to slip away."

heart disease

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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