Astellas treatment offers new hope to a devastated rare disease community

Five years after a gene therapy trial for the fatal muscle disorder XLMTM unraveled amid a string of devastating liver failure deaths, patients and their families are getting a second chance.

Astellas has launched a new clinical trial using a next-gen virus engineered to target muscle more precisely at dramatically lower doses, STAT’s Jason Mast writes. The trial is raising hopes for a safer version of a therapy that once helped some children come off ventilators, walk, communicate, and survive against impossible odds.

Take, for instance, the astonishing early recoveries of boys like Joshua Jacob Gonzalez — but also the field-shaking deaths that chilled the gene therapy sector.

“When the full results of the initial clinical trials are revealed, I believe the world will recognize this work for what it truly is — nothing short of miraculous,” one mother of a son who died of the disease, but who helped fund early research, told STAT. “And still, I hold a measure of trepidation. Concerns remain around liver safety and whether current dosing strategies are sufficient to fully realize the therapy’s potential.”

WASHINGTON

Will Trump oust embattled FDA chief Marty Makary?

President Trump is reportedly preparing to fire Marty Makary, capping a turbulent year atop the FDA. His tenure has been marked by aggressive policy rollouts, internal dysfunction, and mounting political pressure from both the White House and factions within the MAGA movement, STAT’s Lizzy Lawrence writes.

Makary was supposed to give an address at an annual FDA 5K race on Friday, and run with staff. He wasn’t present. A couple of days earlier, as rumors of his imminent departure swirled, STAT asked Makary outside the FDA Law Institute conference on Wednesday how long he plans to stay at the agency.

“Feeling good,” he said, with a thumbs up.

On Friday evening, Trump was asked about the reports he planned to fire the FDA commissioner. “I’ve been reading about it, but I know nothing about it,” he said.

medicare

Alzheimer's drug rollouts fall far below expectations

Two years after Medicare officials warned that new Alzheimer’s drugs could cost the program billions, uptake of the medicines Leqembi and Kisunla have been strikingly muted, STAT’s Bob Herman writes.

Medicare spent just a few hundred million dollars on the drugs through most of 2025 — a fraction of early projections — as patients and physicians grapple with complicated infusion schedules, repeated brain imaging requirements, and bleeding risks. Beyond that, there are lingering doubts about whether the treatments produce meaningful real-world benefits.

"There’s no way to know," one neurologist said. "There’s no way you can judge progress with a drug that slows worsening. You can see people stabilize, but if they get worse at a slower rate, how the hell do you know?"

startups

China-based heart drug startup posts promising data

Just months after emerging from stealth, Braveheart Bio is reporting Phase 2 data suggesting its experimental myosin inhibitor may do more than simply ease symptoms of hypertrophic cardiomyopathy.

The company and partner Hengrui Pharma said the drug, BHB-1893, improved biomarkers, exercise capacity, heart structure, and patient symptoms in a difficult-to-treat subgroup of patients with non-obstructive HCM. The drug could serve as a challenger to Bristol Myers Squibb’s blockbuster heart drug Camzyos.

“We are at an inflection point where there will be advances in cardiovascular [disease] that we want to be on the front end of,” Braveheart CEO Travis Murdoch told STAT late last year.