Patients with GSDIa survive by eating raw cornstarch around the clock, including through the night. The endpoint that won approval was how much less of it they needed.
Some people carry the plaques and never decline. The difference may be whether their microglia crossed one threshold — and stayed on the right side of it.
Asthma is commoner in boys before puberty and in women after. A mouse study proposes the switch runs through male hormones building nerves in the lung — not through hormones acting on immune cells directly.
EGFR-mutant lung cancers escape targeted therapy by amplifying a second driver. SAFFRON blocked both at once — and extended survival against chemotherapy without using any.
Confirming Alzheimer’s biology once meant a PET scan or a spinal tap. PrecivityAD2 does it from blood — and is cleared for symptomatic adults as young as 40.
Device regulation assumes a product behaves the same way every time. Generative AI does not, and the FDA has signalled it is working out what to do about that.
81% sensitivity for cancer, 13.7% for the polyps colonoscopy removes before they become cancer. The second number is what the test cannot do.
A three-year-old company that raised $121 million sold for $820 million upfront. The asset is a drug for 2,500 US patients whose Phase 1b/2a may itself support approval.
Thirty patients, some followed 25 years, 450 blood samples. The ones who progressed were accumulating mutations long before anything showed clinically.
A brief, precisely dosed exposure to a signal that normally suppresses immunity turned natural killer cells into tumour infiltrators — with no genetic engineering, which is what could make it cheap.
Five years, 1,300 patients, 500 enrolled in Johns Hopkins trials — without leaving central Pennsylvania. The trial access is the part that is hard to replicate.
People with almost no body fat develop the metabolic disease associated with obesity. That paradox is what fat tissue failing actually looks like.