Ultragenyx priced Fayuvi, its just-approved gene therapy for a fatal childhood disease, at $3.95 million — reigniting the hard question of how society pays for one-time treatments that carry blockbuster price tags but treat only a handful of patients.
Removing the fallopian tubes during another planned surgery can sharply cut the risk of the deadliest ovarian cancer. The evidence is strong; the challenge is making sure eligible people are actually offered it.
Among people who had both an irregular heartbeat and Alzheimer’s, those on newer anticoagulants declined slightly more slowly — a hint that protecting the brain’s blood supply may help, though the study can’t prove cause.
Some cells begin the self-destruct process, then abort it and go on to rebuild damaged tissue. The finding reveals a hidden engine of regeneration — and a possible clue to why some cancers come back stronger.
A major review confirms that exercise-based cardiac rehabilitation delivers lasting benefits after heart disease. The bigger problem, a companion review finds, is that most eligible patients never take part.
Wernicke encephalopathy, caused by a lack of vitamin B1, can be reversed if caught early — but doctors frequently miss it because the “classic” warning signs usually don’t all show up.
By disabling the antioxidant shield that tumor cells use to survive, the experimental drug RSO-021 lets a cancer’s own toxic byproducts build up until it destroys itself — with promising early results in a hard-to-treat disease.
Injectable peptides rule the obesity market, but they’re hard to turn into pills. Novo’s new alliance with Orbis targets a class of ring-shaped molecules designed to survive the gut.
Using stem-cell-derived “kidney organoids,” scientists showed that high glucose alone can trigger damaging inflammation — and that blocking specific pathways protected the tissue.
Fayuvi, a one-time gene therapy from Ultragenyx, is the first treatment approved for Sanfilippo syndrome type A — a rare disorder that steadily robs children of the abilities they once had.
The maker of Ozempic and Wegovy is deploying Anthropic’s Claude models across its R&D and engineering — the latest sign that Big Pharma sees AI as a core tool, not an experiment.
The blood-brain barrier blocks most large-molecule drugs from reaching the brain. Novartis is buying Sironax’s delivery technology to get antibodies, proteins and gene therapies through it.