For how we live.
I-SPY2.2 tested trastuzumab-deruxtecan plus rilvegostomig before surgery in high-risk HER2-negative breast cancer. In immune-positive tumours 54% had a complete response, and most of them avoided anthracyclines.
In a 98-patient French trial, six-monthly rituximab did not extend remission beyond daily azathioprine in this rare eosinophilic vasculitis — and recipients had more severe infections.
GSK's PD-1 blocker won accelerated US approval on 19 October for previously untreated stage II/III dMMR rectal cancer. In the single-arm AZUR-1 trial, most patients avoided surgery, radiation and chemotherapy.
In 120 older adults with acute myeloid leukaemia, adding midostaurin to two-drug therapy did not raise remission rates overall (72% vs 68%), but a small FLT3-mutated subgroup lived longer.
A single-centre randomised trial found the Bergen 4-day treatment cut obsessive-compulsive symptoms far more than an unguided internet CBT programme, with 61% responding at three months versus 25%.
A 210-youth randomised trial of a telehealth aerobic-exercise program found faster symptom recovery at three months than stretching — an edge that disappeared by six months and held only for recent injuries.
In 143 people with poorly controlled type 1 diabetes, a system that prompts injections through glucose-sensor alerts was non-inferior to standard monitoring — and acting on the alerts drove the benefit.
A six-site randomised trial of point-of-care high-sensitivity troponin cut emergency-department length of stay by 47 minutes, with no change in 30-day heart attacks or cardiac deaths.
In 1,429 high-risk breast-cancer patients, a 16-dose regimen covering the internal mammary nodes matched the standard 25-dose course for recurrence and caused far less severe skin damage.
In 1,095 high-risk abdominal-surgery patients, targeting each person's own cardiac index during the operation left the rate of major complications essentially unchanged — 44.3% versus 46.9%.
The FDA approved Tecentriq on 8 October, with chemotherapy, for Stage III mismatch repair deficient colon cancer after resection. In ATOMIC, adding it cut disease-free survival events, hazard ratio 0.50.
In a 17-person phase 2a trial, once-weekly inhaled SPL84 lifted lung function in up to 70% of treated patients with the 3849+10 kb C→T mutation — a first reported clinical signal for an RNA-targeted inhaled therapy.
LOTIS-9 tested loncastuximab tesirine plus rituximab in unfit and frail older adults with untreated DLBCL. Complete responses reached 53% and 42%, but severe side effects, including 11 deaths, ended the trial early.
SLOWH, a 102-patient randomised trial, compared cutting flow first, oxygen first, or both at once when weaning critically ill adults off high-flow nasal cannula. Success rates of 94%, 81% and 77% did not differ.
A 60-woman phase 2A trial found the dual PPAR agonist reduced liver fat by nearly 15 percentage points more than placebo over 24 weeks — an early, small signal in a group usually left out of fatty-liver drug trials.
In the 17,701-person E.mbrace trial in adults 60 and older, the nine-valent ExPEC9V vaccine showed 14.63% efficacy, with a confidence interval spanning zero. It stopped early for futility; development is discontinued.
A detailed safety analysis of the 12,254-patient OCEANIC-STROKE trial found no significant rise in major bleeding with the factor XI blocker, though lower gastrointestinal bleeding was more frequent.
A prespecified analysis of 227,502 veterans who contacted the Veterans Crisis Line found suicide mortality of 0.29% among caring-letter recipients versus 0.27% in historical controls — no measurable difference.
In a 180-infant phase 1/2 trial, a live-attenuated intranasal RSV vaccine raised neutralising antibodies in two-thirds or more of RSV-naive babies but barely budged those already exposed. Sanofi funded the study.
Teva's subcutaneous depot is built to avoid the post-injection sedation that hobbled the first olanzapine shot. It cleared the FDA on 8 October; in the phase 3 SOLARIS trial it beat placebo on symptoms at eight weeks.
In the phase 3 VERIFY trial, 77% of patients on the hepcidin-mimic drug avoided phlebotomy over weeks 20–32 versus 33% on placebo, and far more kept their haematocrit under control.
The pill joins trastuzumab and pertuzumab as maintenance after induction. In the 654-patient HER2CLIMB-05 trial it stretched median progression-free survival to 24.9 months from 16.3, a hazard ratio of 0.64.
The phase 3 RT CHARM trial found a three-week course of radiation after mastectomy was no worse than the standard five-week course for reconstruction complications, at 12.3% versus 10.2%.
Rhapsido becomes the first drug labelled for the itchy welts raised by scratching or pressure. In the RemIND trial, 29.3% on the pill cleared the provocation test at week 12 versus 14.0% on placebo.
In 204 patients already on standard therapy, pressing a seed on one ear point three times daily left 56.9% clear or almost clear at 8 weeks versus 35.3% with a sham point — a single-centre result needing replication.
The phase 2 ROXAMI trial gave the oral HIF stabiliser roxadustat to 158 STEMI patients after stenting. Infarct size at 30 days was no smaller than with standard care. Later remodelling findings were exploratory.
RINGSIDE phase 2 tested three doses of oral varegacestat in 42 adults with progressing desmoid tumours. The lowest daily dose gave the deepest shrinkage and was carried into phase 3. No comparator arm.
In a 28-patient single-arm phase 2 study, disitamab vedotin with tislelizumab produced a complete response in 73% of evaluable HER2-positive bladder cancers that could not be resected. There was no comparator.
ESO-T01 builds cancer-killing T cells inside the body, skipping the cell harvest and chemo prep normal CAR-T needs. All four responded — but the cells faded, and median progression-free survival was 4 months.
A phase 1/2 trial gave mosunetuzumab plus polatuzumab to 101 older, unfit or frail people with untreated large B-cell lymphoma. The end-of-treatment response rate was 61.4%, with a complete response in 56.4%.
A single-arm phase 2 trial added glofitamab to pola-R-CHP in 41 people with newly diagnosed B-cell lymphoma. The best complete-response rate was 95%, cytokine release stayed grade 1, and no neurotoxicity was reported.
The agency is describing the trial design it is weighing for ibogaine, a psychedelic with serious cardiac risks, as HHS funds the first federal studies in opioid use disorder and PTSD. Comments close 20 November.
A randomised trial enrolled 3,259 underserved people referred for lung cancer screening. Adding free medication to standard advice did not raise quit rates; adding financial incentives roughly doubled them — to 8.8%.
A Toronto cohort reports that an eight-day antiviral protocol prevented or cleared hepatitis C in all 116 recipients of organs from infected donors, with survival matching recipients of uninfected organs.
In the Nordic SWEDEGRAFT trial, the no-touch vein-harvesting technique left the rate of death, heart attack, or repeat procedures at 19.8% versus 20.4% through seven years — a difference indistinguishable from chance.
In the industry-funded evERA trial, giredestrant plus everolimus delayed progression of ER-positive breast cancer — but the clearest benefit was in ESR1-mutated tumours, and the endpoint was a surrogate, not survival.
Thirteen years on, the independent DiAPREV-IT trial still shows no overall effect from GAD-alum. A 27-child genetic subgroup saw onset delayed — but the other subgroup got worse.
A draft NIH policy out for comment would require researchers to share plain-language summary results with clinical research participants for all NIH-supported studies, unless justifiably not appropriate.
The UV-free TransVitD patch raised vitamin D in animals and lab-grown skin. In a randomised trial of 108 volunteers it was safe but did not lift blood levels beyond what a small oral dose would.
AbbVie's once-daily pill cleared the FDA on 25 September as a new molecular entity. Across three phase 3 TEMPO trials it beat placebo in early Parkinson's and as an add-on to levodopa.
In the phase 3 SYNCHRONIZE-2 trial, the glucagon/GLP-1 dual agonist beat placebo on weight over 76 weeks. The effect was solid but smaller than the class leaders manage, and the maker funded it.
Two phase 3 trials put the oral drug safiglipron ahead of placebo and non-inferior to dapagliflozin on HbA1c. Both ran only in China, were maker-funded, and showed dose-dependent gut side effects.
In the phase 3 UKALL14 trial, one cycle of the T-cell drug nelarabine after induction was well tolerated but left event-free survival essentially unchanged in adults aged 25 to 65.
A randomised Asian trial in 130 patients found no efficacy gap between the two drug regimens for active lupus nephritis, but serious infections and all three deaths fell in the mycophenolate group.
ROAM/EORTC-1308, an independent phase 3 trial, randomised 157 patients after complete brain-tumour removal; adjuvant radiotherapy improved five-year disease-free survival, but the benefit's confidence interval was wide.
RESOLVE randomised 880 treatment-naive, non-cirrhotic patients to a shorter sofosbuvir–velpatasvir course; cure rates were non-inferior to the 12-week standard, with implications for the cost of elimination programmes.
The PLUSS trial put a steroid directly into the lungs of 1,059 infants born before 28 weeks. Survival free of chronic lung disease barely differed from surfactant alone.
A single-centre, placebo-controlled trial found targeted onabotulinumtoxinA injections cut dystonia-related foot pain without harming movement. The effect was large, but the trial randomised only 33 people.
Final results of the phase 3 IMpassion050 trial show adding the immunotherapy atezolizumab to standard HER2-targeted treatment did not improve event-free or disease-free survival.
The phase 3 CLL6 RESIDUUM trial extended progression-free survival by about 22 months in patients left with residual disease, but overall survival did not differ and the drug caused more infections.
The CENTERSTONE trial found a single dose lowered lab-confirmed spread to household contacts by about a third, but spread that made contacts ill was not significantly cut, and resistant virus emerged in some patients.
In the Scandinavian phase 3 ASAC trial, daily aspirin failed to improve disease-free survival after treatment of colorectal cancer liver metastases — and overall survival was worse on the drug.
ACHIEVE-4 found the non-peptide GLP-1 pill non-inferior to insulin glargine for major cardiac events over two years, with less severe hypoglycaemia but far more gastrointestinal upset. Lilly funded it.
Ketamine cuts suicidal thinking fast but briefly. A 50-person trial gave low-dose buprenorphine afterward and saw the effect hold — an early, single-centre result in a high-risk group.
In a 366-child phase 2b trial, the live attenuated nasal vaccine BPZE1 raised the mucosal antibody current shots miss, and did not blunt a co-given Tdap. The maker funded the study.
ZUPREME 1 tested a once-weekly amylin analogue that works differently from GLP-1 drugs, finding up to 9.4% weight loss at 28 weeks and gastrointestinal side-effects close to placebo. Zealand Pharma funded it.
Merck's sotatercept gained an FDA efficacy supplement on 21 September. In the phase 3 ZENITH trial, a composite of death, transplant or hospitalisation hit 17.4% on the drug versus 54.7% on placebo.
In TRIUMPH-1, a triple hormone-receptor agonist produced a 25% average weight loss at the top dose over 80 weeks, and also eased knee pain and sleep apnoea. The trial was funded by Eli Lilly.
The FDA approved PTC Therapeutics' sepiapterin in July 2025. In the phase 3 APHENITY trial, responders saw a 63% fall in blood phenylalanine over six weeks against roughly no change on placebo.
In the phase 3b STRIDE trial, people with peripheral artery disease and type 2 diabetes walked 13% farther at 52 weeks than on placebo (treatment ratio 1.13; p=0.0004). The gain is modest; Novo Nordisk funded it.
A randomised trial at eight South Korean hospitals found pulse methylprednisolone no better than a non-pulse regimen for surviving an acute exacerbation of idiopathic pulmonary fibrosis.
A Swiss investigator-led trial tested whether isolating the back wall of the left atrium on top of pulsed-field pulmonary vein isolation cuts arrhythmia recurrence. The primary answer was no.
In the phase 2 KRAKEN trial, muvalaplin lowered lipoprotein(a) by 47.6% to 85.8% over 12 weeks on an intact-particle assay. Eli Lilly funded it, and the trial was not built to measure heart attacks or strokes.
In the GSK-funded phase 3 MATINEE trial, the anti-IL-5 antibody lowered moderate or severe exacerbations from 1.01 to 0.80 a year — but symptom and quality-of-life endpoints showed no significant benefit.
In a phase 2 trial in people whose hidradenitis suppurativa had already failed anti-TNF drugs, two lutikizumab doses beat placebo at 16 weeks. A third did not, and the placebo response was high.
In the phase 3 LIBerate-HR trial, lerodalcibep lowered LDL cholesterol 56.2% more than placebo at 52 weeks on top of statins. It won approval in 2026; no outcome trial has yet reported.
Eli Lilly's oral Inluriyo gained an FDA efficacy supplement on 18 September. In the phase 3 EMBER-3 trial, adding abemaciclib lifted median progression-free survival to 9.4 months from 5.5.
The oral histone deacetylase inhibitor cleared the FDA in March 2024. In the phase 3 EPIDYS trial, boys on givinostat still lost function over 72 weeks — just less of it than those on placebo.
A single-surgeon randomised trial of 180 knees found small but statistically supported gains for robot-assisted functional alignment over conventional mechanical alignment at two years.
In EMPACT-MI, the SGLT2 inhibitor missed its primary endpoint: 8.2% of patients had a heart-failure hospitalisation or died versus 9.1% on placebo (hazard ratio 0.90; p=0.21). Boehringer and Lilly funded it.
Emcitate cleared the FDA on 28 September as a new molecular entity. In an open-label trial of 45 patients, the drug lowered the high serum T3 that drives MCT8 deficiency by about two-thirds.
In the DIABIL-2 trial, low-dose interleukin-2 hit its immune target in people newly diagnosed with type 1 diabetes, yet failed to preserve insulin-producing cells at one year.
Bristol Myers Squibb's mavacamten gained an FDA efficacy supplement on 30 September. In the pivotal EXPLORER-HCM trial, 37% on the drug met the primary endpoint versus 17% on placebo.
AMPLIFIED enriched for early rheumatoid arthritis patients thought to respond best to abatacept, then compared it head-to-head with adalimumab. At week 24 the two drugs were indistinguishable.
A phase 2b trial in 4936 Gambian children missed its primary endpoint, but ETVAX cut moderate-to-severe ETEC diarrhoea by 80.6% once parasite co-infections were excluded.
In the pragmatic BEAT Tuberculosis trial, an all-oral six-month regimen cured 86.1% of patients with rifampicin-resistant TB — statistically non-inferior to the nine-month standard, with similar safety.
Rezzayo's once-weekly echinocandin can now be used in adolescents with candidaemia who have few other options. The expansion rests on adult data and modelling, not on studies in children.
In the publicly funded SWOG S1600 trial, specialised immunonutrition before and after radical cystectomy left 30-day complication rates essentially unchanged: 62% versus 58% on a standard drink.
The academic MATRix/IELSG43 trial found autologous transplant lifted 3-year progression-free survival to 78% from 51%, but at the cost of more toxicity and more treatment-related deaths.
A phase 1 trial in 288 Chinese children found the experimental injectable vaccine well tolerated and able to raise antibodies. It measured immune response, not whether it prevents disease.
A Shionogi-funded randomised trial found that giving household contacts a five-day course dropped the rate of symptomatic Covid from 9.0% to 2.9%, with no severe cases in either group.
The publicly funded E4512 trial found two years of crizotinib after surgery did not prolong disease-free survival against observation, with a hazard ratio of 1.08 and no safety upside.
In the investigator-led Danish DALIAH trial, pegylated interferon-alfa and hydroxyurea produced similar molecular responses by intention-to-treat, but far more patients stopped interferon.
In the LOSE-AF trial, an eight-month low-calorie programme cut weight by 9.7% in older adults with AF. It did not change their symptoms, their AF burden, or their need for further rhythm treatment.
Cleared on 10 July under NDA 218579, the Provepharm agent fluoresces in near-infrared light to map vessels, perfusion and lymph nodes during surgery. Efficacy rests on studies of an earlier formulation.
Approved on 24 June under NDA 220436, the Avyxa Holdings product is a ready-to-infuse form of the NK-1 blocker fosaprepitant, cleared on studies of an earlier intravenous version of the drug.
Added to an SGLT2 inhibitor, the aldosterone-synthase inhibitor cut office systolic pressure 7.5 mm Hg and albuminuria about a quarter in 59 adults with CKD. Kidney function dipped slightly; the maker funded it.
A pooled analysis of four trials found depemokimab roughly halved exacerbations, yet it did not significantly improve asthma control or reach the threshold for a meaningful quality-of-life gain.
In the phase 3 Bax24 trial, the aldosterone-synthase inhibitor cut 24-hour ambulatory systolic pressure 14.0 mm Hg more than placebo over 12 weeks. AstraZeneca funded it, and three patients developed high potassium.
There is still no licensed Lyme vaccine. In a phase 2 trial of VLA15, a second yearly booster revived antibody levels against all six OspA serotypes, with more sore arms but no new safety signals.
Gilead's phase 3 trial in 539 patients was terminated after the CD47 antibody failed to improve remission or survival and caused more fatal adverse events than azacitidine alone.
There is no approved drug for methamphetamine use disorder. In 339 patients treated in ordinary clinics, a generic antidepressant cut use by about two days a month more than placebo, with more dropouts from side-effects.
In the industry-funded trial, young responders who kept taking the dopamine D1 blocker relapsed less than those tapered to placebo. The effect was clear in children but not in the small adult group.
Ruxolitinib cream beat a placebo cream for cutaneous lichen planus over 16 weeks, but the phase 2 trial randomised only 64 patients and was funded by the drug's maker.
Regeneron's activin A antibody, brand name Pasatru, cleared the FDA on 19 August. In the pivotal 63-patient trial, new abnormal-bone lesions fell by 90% or more against placebo over 56 weeks.
The Scholar Rock antibody cleared the FDA on 11 September. In the phase 3 SAPPHIRE trial, apitegromab plus standard therapy beat placebo by 1.8 points on a motor-function scale at 12 months.
The final analysis of Roche's phase 3 trial found the anti-TIGIT antibody did not significantly extend progression-free or overall survival when added to atezolizumab in 521 patients.
Two-year blinded data and open-label follow-up from the ELATIVE trial show the drug kept improving liver blood tests in primary biliary cholangitis — but the endpoint is still a surrogate, and the trial is industry-run.
In the company-run CONDOR trial, brolucizumab injections outperformed panretinal laser for proliferative diabetic retinopathy over 54 weeks — while intraocular inflammation was far more common in the drug arm.
In 3,000 patients eligible for blood thinners, the device maker's trial found left atrial appendage closure noninferior to anticoagulant drugs, and superior for non-procedure-related bleeding at three years.
LOGICAL randomised 1,840 unresponsive adults in Australia, New Zealand and Ireland to conservative or liberal oxygen in intensive care. Favourable outcome at 180 days was 38.2% against 39.7%.
In the phase 3 INDIGO trial, weekly obexelimab roughly halved the risk of a flare in this rare fibroinflammatory disease and spared patients steroids — a positive result in a condition with no approved drug.
In 410 German women, 68% who had liposuction met the pain-reduction target at 12 months versus 8% on conservative therapy. Serious adverse events were higher after surgery.
The VITAL trial randomised 2,508 patients aged 50 and older to propofol-based or inhaled anaesthesia and found no difference in days alive and at home at 30 days, or in death, delirium or major complications.
The TRACK trial was stopped early for futility. The blood thinner failed to reduce a composite of cardiovascular events, while major bleeding rose by half. Bayer, which makes the drug, was a listed collaborator.
In the phase 3 TenCRAOS trial, intravenous tenecteplase given within 4.5 hours did no better than aspirin at recovering sight, and carried more bleeding — including one fatal brain haemorrhage.
The NIH-funded STArT trial in children and young adults was halted for futility: time to crisis resolution was 60.8 hours with arginine versus 65.8 with placebo, well within the range of chance.
SOLARIS, a publicly funded phase 3 trial, tested vitamin D3 added to chemotherapy after a promising phase 2 signal. Progression-free survival was 11.8 versus 10.3 months — not a significant difference.
A double-blind Danish trial gave the drug or placebo during resuscitation at 21 hospitals. Restored circulation occurred in 39% versus 37% — no meaningful difference — and bicarbonate raised blood alkalosis and sodium.
In the SNAP platform trial, cefazolin was non-inferior to flucloxacillin or cloxacillin for 90-day survival in methicillin-susceptible S. aureus bacteraemia, and caused notably less acute kidney injury.
In the phase 3 SWOG/NRG S1914 trial, adding atezolizumab to stereotactic radiotherapy for high-risk, inoperable early lung cancer left 2-year survival at 82% in both groups and caused more serious side effects.
In the industry-funded ADVANCE OUTCOMES trial, the once-daily prostacyclin-pathway drug more than halved the risk of a first worsening event, but drove far more treatment discontinuations for side-effects.
The PROPOSITIS trial put babies on high-flow oxygen face-down or on their backs. Escalation of care was 15% versus 21% — a difference that did not reach significance, and the authors call the study inconclusive.
In GASTRIC-PICU, 4,700 ventilated children were randomised to routine gastric residual checks or none. Dropping the checks was non-inferior for survival and ventilator-free days, and improved nutrition.
Ceperognastat, an oral OGA inhibitor, showed no benefit on the primary cognitive endpoint at 100 weeks. The higher dose progressed 32% faster than placebo and had more serious adverse events.
REPLENISH randomised 381 patients with relapsed polymyalgia rheumatica. Sustained remission at 52 weeks reached about 41% on secukinumab versus 20.4% on placebo. Novartis funded the trial.
In the 1,658-person PUSH trial, a behavioural programme with financial incentives raised urine volume but left symptomatic stone recurrences unchanged over two years — 19% against 20% with usual care.
In the phase 2 Vibrance-1 trial, alixorexton lifted mean sleep latency from about 2 minutes on placebo to 24 or more on the drug. The six-week study was funded by the manufacturer.
In the SOLARIO trial, patients who got local antibiotics at surgery did no worse on a short systemic course than a long one, and reported far fewer side effects. The comparison was independently funded.
In a single-arm trial, children with vanishing white matter given guanabenz kept walking longer than matched historical controls. The design was uncontrolled and hallucinations were common early on.
In the publicly funded EPoS II trial, waiting five years before the next colonoscopy was no worse than the recommended three at an interim look, with near-identical cancer rates. Ten-year data are still to come.
A 40-person Harvard trial aimed accelerated magnetic stimulation using each patient's own connectivity scan. Depression scores fell further than with standard scalp measurement, though the trial was small.
In the French GETUG AFU 18 trial an 80 Gy dose beat the standard 70 Gy on 10-year progression-free survival (83.6% vs 72.2%), but the advantage at the prespecified 5-year mark was far smaller.
Varespladib, given on top of antivenom in a randomised trial in India and the USA, did not beat placebo. But it reached patients seven hours after the bite, and the authors say that timing may be the story.
TEMPLE, an AbbVie-funded phase 3b trial, is the first direct test of the CGRP blocker atogepant against topiramate — a long-used preventive many patients cannot tolerate.
SINGLE-AF randomised 1,803 South Koreans with borderline stroke risk to a direct oral anticoagulant or nothing. The drug lowered a composite of stroke, bleeding and death at two years, from 1.5% to 0.5%.
In PHOENYCS GO, dapirolizumab pegol lifted the share of lupus patients reaching a composite response at 48 weeks to 50% from 35% on placebo — a real but modest 14.6-point gain.
In the investigator-led CAVIAR trial, adding the PCSK9 inhibitor alirocumab to a statin after heart transplant more than halved LDL cholesterol but did not slow coronary plaque growth at one year.
An 840-child US trial gave the antibiotic to toddlers with moderate-to-severe wheezing in the emergency department. Symptom scores were no better than placebo, and the study was stopped for futility.
The Essilor-designed HAL lenses cut two-year myopia progression by 71% versus ordinary glasses in 159 American children. The company employs the study's authors, and the result backs the device's US clearance.
A direct final rule swaps 'animal' for 'nonclinical' across five parts of FDA's drug regulations. It adds no requirements, but signals a shift the agency began with monoclonal antibodies.
In deficient populations, vitamin A supplements cut child deaths by 12% — among the best-evidenced global-health interventions. In the well-nourished, added vitamin A does no good and can do harm.
Cellular senescence is a well-evidenced driver of ageing in animals. The first human trials of senolytic drugs that clear these cells are real but tiny, early, and nowhere near proving longer healthy life.
In the TESEO trial, benznidazole for 30 days matched the 60-day standard for clearing the parasite and caused fewer side effects, pointing to a gentler regimen for a neglected disease.
Open-label follow-up of the phase 3 LUNA3 trial shows rilzabrutinib's platelet responses were durable and sometimes appeared in people who had not responded at first — but a minority reached the mark.
In people with no heart disease, aspirin's benefit was small or absent and bleeding rose across three large trials. US guidance now advises against starting it at 60 or older.
In the CREST-2 trials, stenting or surgery added to intensive medical treatment left cognition no better at four years than medical treatment alone in people with severe asymptomatic carotid stenosis.
ASPREE randomised 19,114 healthy older adults to low-dose aspirin or placebo. It made no difference to disability-free survival, and modestly raised deaths and major haemorrhage.
At one year, 79% of newly diagnosed patients given early infliximab plus an immunomodulator were in steroid-free, surgery-free remission, versus 15% treated conventionally.
In the 2,137-participant PrEggNut trial, mothers who ate a diet rich in eggs and peanuts through pregnancy and breastfeeding had infants with the same allergy rate as the comparison group.
An independent 2026 meta-analysis of AAV micro-dystrophin trials found small, mostly uncontrolled functional benefits — largest in the youngest boys — set against serious harms including rare deaths.
A small 1990s trial hinted selenium supplements cut cancer, launching a supplement boom. The much larger SELECT trial found no benefit — and signals of harm, including more type 2 diabetes.
Diets rich in colourful fruit and vegetables track with less cancer, so supplement makers bottled beta-carotene. Two large trials in smokers found the pills raised lung cancer and death — the opposite of the hope.
In the ProtecT trial's patient-reported data, surgery hit urinary control and erections hardest and radiotherapy the bowel, while monitoring let function fade slowly. Cancer deaths were the same.
A landmark trial found men could safely delay repair of a minimally symptomatic groin hernia. Over the following decade, though, most crossed over to an operation as symptoms grew.
A widely cited cohort found people with diabetes on metformin outlived matched non-diabetic controls. It fuelled the TAME anti-ageing trial — but the human signal is observational, and TAME is not running.
The first randomised head-to-head trial of the two obesity drugs gave 751 adults the maximum tolerated dose of each for 72 weeks. Tirzepatide cut weight by 20.2%, semaglutide by 13.7%.
Two large trials, ETHOS and IMPACT, found adding an inhaled steroid to a two-bronchodilator inhaler lowered exacerbation rates in COPD — at the cost of more pneumonia.
In 370 nulliparous women, instilling mepivacaine into the uterus before IUD placement lowered pain by about 15 mm on a 100-mm scale versus saline. It is a real effect, not a cure for the procedure.
RelieVRx is an authorised virtual-reality therapeutic for chronic low back pain. Its trials show real improvement — but in a 1,067-person study the edge over a placebo headset was 0.4 points on a 10-point scale.
In the LATA trial across four countries, 0.9% of teens on long-acting cabotegravir–rilpivirine lost viral suppression by 96 weeks, versus 6.4% on daily tablets — a superiority result.
In FINEARTS-HF, 6,001 patients with an ejection fraction of 40% or more had fewer worsening-heart-failure events on finerenone. Cardiovascular deaths were not lower.
In a 230-patient randomised trial, self-guided app hypnotherapy produced a 33% pain response versus 48% with an in-person therapist and 22% with education — falling short of non-inferiority.
In 709 children with high-risk B-cell ALL, swapping two chemotherapy cycles for the T-cell-engaging antibody lifted four-year event-free survival to 83% from 70% and sharply cut serious infections.
SOLARIO randomised 500 patients after orthopaedic infection surgery to short or long systemic antibiotics. With a local-antibiotic carrier in place, the short course was noninferior and caused fewer side effects.
Amycretin, a single molecule that activates both the GLP-1 and amylin receptors, produced up to 24.3% weight loss over 36 weeks in a 125-person study. The primary goal, though, was safety — not weight.
Abaloparatide reliably raises bone density, so it seemed a natural candidate to help fractures knit. A randomised trial found no benefit — and healing numerically favoured placebo.
In HIMALAYA, 305 people with ALS took the RIPK1 inhibitor SAR443820 or placebo; function fell almost identically in both, and the sponsor is halting the drug in ALS.
Obicetrapib cut Lp(a) by 37% in high-risk patients, a pooled analysis finds, alongside a similar drop in LDL. It reaches a lipid the costly new injectables target, if far less deeply.
LITESPARK-022 cut the risk of recurrence by 28% when belzutifan was added to pembrolizumab after surgery. Survival was no different, and severe side effects nearly doubled.
External validation of the PAANTHER decision algorithm in 277 children across four African countries recorded 93.3% negative predictive value and 97.4% sensitivity against a composite reference standard.
In a 142-patient phase 3 trial, 37% reached complete remission on the antibody obinutuzumab versus 6% on tacrolimus at two years. A key kidney-function endpoint was not met, tempering the result.
In ESSENCE, the first 800 patients with biopsy-confirmed fatty-liver disease were analysed at week 72. Steatohepatitis resolved in 62.9% on semaglutide versus 34.3% on placebo — an interim look at an ongoing trial.
Two trials found low-dose colchicine cut cardiovascular events, and the FDA approved it for heart risk in 2023. Then the largest trial found nothing, leaving the question genuinely unsettled.
A new analysis of the REDEFINE 1 trial reframes obesity treatment around reaching a normal BMI and waist size, not just percentage lost. The combination beat semaglutide alone on that measure.
Adding the immunotherapy to perioperative chemotherapy improved overall survival in resectable gastric and junction cancer — crossing the significance line the interim analysis had missed.
Cobenfy, approved in the US in September 2024, cut symptom scores more than placebo in two five-week trials — without the weight gain and movement effects of older drugs, but with cholinergic side effects.
Testosterone raises desire mostly in men whose levels are genuinely low, and the effect is modest. In the largest trial, the size of the hormone rise, not a threshold, tracked with benefit.
In the phase 3 THRIVE trial, veligrotug shrank bulging eyes in 70% of patients versus 5% on placebo. It is the first serious challenger to the only drug approved for the condition.
In a 711-patient phase 2b trial, the myeloperoxidase inhibitor mitiperstat did not improve symptoms or walking distance in heart failure with preserved or mildly reduced ejection fraction.
A daily testosterone-and-progestin gel is in a 52-week efficacy trial in 462 couples. A non-hormonal pill cleared its first safety study in 16 men. Neither is approved.
A child with a suspected concussion should be removed from play and not return the same day. But prolonged strict rest is no longer advised, and light activity within days can speed recovery.
The largest study, comparing siblings to strip out family confounding, found no link between paracetamol in pregnancy and autism or ADHD. Guidelines still call it the preferred painkiller when one is needed.
Guidelines now ask for an objective test before an asthma label is fixed to a child, and inhaled steroids remain the mainstay of control. Their benefit is real; so is a small, mostly temporary effect on growth.
The main randomised trial found babies who fed themselves were no slimmer, no more iron-deficient and no more likely to choke than spoon-fed babies — provided parents were coached to offer safe, iron-rich foods.
A single-centre randomised trial found that three cycles of intravenous immunoglobulin on top of high-dose steroids improved muscle disease faster and further — in just 44 patients, at one Dutch hospital.
Two large African trials gave anaemic pregnant women intravenous ferric carboxymaltose or oral iron. Neither found less anaemia near term, or heavier babies, with the infusion.
One drug, dapoxetine, is purpose-approved for it, and not in the US. An umbrella review of 65 trials found the SSRIs and anaesthetics that lengthen latency rest on weak evidence.
For the pelvic-pain syndrome that affects many men, Cochrane reviews find alpha-blockers, antibiotics and anti-inflammatories each help only a little. Acupuncture and shockwave therapy have the firmer support.
Epigenetic clocks estimate a 'biological age' from DNA methylation. A 2022 study found technical noise alone could shift the result by up to 9 years between replicates of the same sample.
A 4,153-woman trial found progesterone did not raise live births overall. The benefit concentrated in women who were bleeding and had had three or more previous miscarriages.
In a phase 3 trial, tambotatug pelitecan extended median survival to 13.3 months versus 9.4 on standard chemotherapy — but the trial was open-label and run entirely in China.
Adding pembrolizumab plus maintenance olaparib to first-line chemotherapy improved progression-free survival — but pembrolizumab added without olaparib did nothing at all.
In PROPEL 3, children on once-daily infigratinib grew 1.74 cm/year faster than those on placebo over 52 weeks. It is a pill, not an injection — but the endpoint is growth speed, not long-term health.
The only FDA-approved drug for the penile-curvature disorder straightens the average man's bend only modestly more than placebo. Surgery is more definitive, and most oral remedies are unsupported.
A 1,784-child randomised trial in Uganda found one-fifth and one-half doses of the 17DD vaccine matched the full dose for initial immunity — evidence for dose-sparing in young children when supplies run short.
AIROPLANE enrolled 1,818 newborns at 32 to 35 weeks across 26 Australian hospitals. Starting with 30% oxygen instead of room air left the same share still on respiratory support leaving the delivery room.
TRIC-I-HF randomised 360 older patients with severe tricuspid regurgitation. Adding transcatheter repair to medical therapy cut a composite of death or heart-failure hospitalisation through three years.
A 1,695-patient trial tested whether metoprolol helps people with COPD and no cardiovascular indication. It missed its primary endpoint — but, unlike an earlier trial, showed no signal of harm.
In QWINT-1, fixed-dose weekly efsitora cut HbA1c about as much as daily glargine in 795 insulin-naive adults — with a lower rate of significant hypoglycaemia and a quarter of the dose changes.
In a 258-patient phase 3 trial, the oral drug reduced transfusion needs in about 30% of adults with transfusion-dependent thalassaemia, versus 13% on placebo.
Randomised trials show acute care delivered at home costs less, uses fewer tests and readmits fewer patients — while making little or no difference to whether they survive.
Sensors and reminder inhalers get patients to take more doses — one trial lifted adherence from 30% to 84%. That this translates into meaningfully better control is real but a smaller effect.
In an 81-child trial, vosoritide raised annualised growth velocity by 2.33 cm a year against placebo in hypochondroplasia, which has no targeted treatment. The endpoint is growth speed, not final height.
Two trials of oveporexton, an orexin receptor agonist for narcolepsy type 1, met their wakefulness endpoint and cut cataplexy sharply. Side effects were near-universal.
In the LAIS trial, more patients given intravenous loberamisal recovered with little or no disability at 90 days — a rare positive result in a field littered with failed neuroprotectants.
Tozorakimab, which blocks interleukin-33, lowered the rate of moderate or severe COPD exacerbations by about a third against placebo, without an eosinophil requirement to qualify.
A postpartum smoking programme and a safe-sleep video series each failed the question they were designed to answer. What they reported next is where the interpretation gets difficult.
One found doctors stopped using it as shifts got busier. The other, in 9,691 Kenyan patients, found it safe but no better than the electronic record alone. Neither supports deployment.
A Polish study found endoscopists' unaided polyp-detection fell after AI arrived. A Norwegian trial that tracked doctors before, during and after AI use found no such deskilling.
OSPREY implanted every participant, then randomly decided whose device was switched on. That design answers a question most implant studies leave open, and it has limits the authors state plainly.
AD109 cut the apnoea-hypopnoea index by four events an hour more than placebo over 26 weeks. It did not improve fatigue, and one in five participants stopped because of side effects.
In a 15-patient phase 1 trial, TX103 cells delivered directly into the brain were tolerated and median survival reached 19.1 months — but this is early, single-arm evidence with no control group.
An arthritis antibody and a diabetes drug were given to depressed patients in 2026 trials. Both were designed to probe a mechanism rather than prove a treatment, and reading them as results would be a mistake.
Two 2026 trials point the same direction: the hardware in depression treatment is mature, and the remaining gains are in precision and in what the treatment costs the patient's memory.
A six-month regimen for drug-resistant tuberculosis matched the standard of care in South Africa. Four days of antibiotics matched eight in African children hospitalised with severe pneumonia.
4CMenB had shown promise against gonorrhoea in observational data. Randomised, its efficacy was minus 0.5%. In the same field, a once-weekly oral HIV regimen met non-inferiority at 48 weeks.
Zenagamtide was tested as a weekly injection and a daily pill in type 2 diabetes. Both cut HbA1c against placebo at 36 weeks, and the oral version's side effects rose steeply with dose.
Satralizumab reduced eye-bulging and inflammation in thyroid eye disease across the twin SatraGO phase 3 trials, but the primary proptosis endpoint reached statistical significance in only one of them.
Tranexamic acid cut postpartum haemorrhage in placenta praevia. Dapagliflozin nearly halved acute kidney injury after cardiac surgery. The effect sizes are very different, and so is how much to trust them.
The two most-prescribed respiratory antivirals sit at opposite ends of the evidence. One shortens flu by less than a day on average; the other cut serious outcomes sharply — but only in the group at real risk.
A single infusion of a pan-serotype monoclonal antibody cut viraemia and shortened fever in a 250-patient phase 2 trial in India — the first therapy to show preliminary efficacy against wild-type dengue in people.
MariTide antagonises the GIP receptor that tirzepatide switches on, yet in 592 adults it drove 12–16% weight loss over a year — and it is dosed once a month, not once a week.
A real condition with a formal case definition, a measurable population-level burden, and — so far — no treatment proven to reverse it. What the studies do and do not establish.
EECOH-1 randomised 211 adults and found the weekly GLP-1 cut HbA1c up to 1.56 points more than placebo over 24 weeks — competitive numbers from a short, single-country, company-run trial.
SERENA-6 changed therapy the moment a resistance mutation appeared in the blood, not when tumours grew. It nearly doubled time to progression — and won camizestrant FDA approval in 2026.
A small single-arm trial of the PD-1/CTLA-4 antibody cadonilimab with lenvatinib reported a 40.6% response rate in previously treated advanced endometrial cancer.
Sepsis is the body's own response to an infection turning against its organs. It kills an estimated 11 million people a year, and the evidence keeps pointing to how fast it is caught and treated.
EVAOLD tested whether stress imaging could safely spare patients aged 80 and over an invasive procedure. It was stopped early for futility, and the selective strategy did not prove non-inferior.
A Danish stepped-wedge trial gave endoscopists automated feedback on their technique after every procedure. Adenoma detection rose from 43.4% to 48.6% — a different tool from real-time polyp AI.
In the 172-patient PARADIGM trial, azacitidine plus venetoclax more than doubled median event-free survival versus induction chemotherapy — but it was a phase 2 study not powered for overall survival.
A blood test after surgery can detect tumour DNA too faint to see on a scan. In one trial it let colon-cancer patients safely skip chemotherapy — but the evidence is still narrow.
Stargardt disease, the most common inherited macular dystrophy, has no approved treatment. In the 50-patient TEASE-1 trial, gildeuretinol slowed atrophic lesion growth by a fraction of a millimetre a year.
In an early-phase trial of 136 previously treated patients, daraxonrasib produced responses in 31–37% depending on dose — but 54% had a serious side effect, and this is a phase 1–2 study.
In the Ket-BD trial, four IV ketamine infusions over two weeks lowered depression scores more than an active placebo, with no manic switches observed in 63 analysed patients.
In a 92-patient Greek cohort, the long-acting PTH replacement normalised calcium and phosphate and freed 58% of patients from calcium and vitamin D pills — echoing the trials, over a short follow-up.
In the NSABP B-59 trial, adding the immunotherapy to neoadjuvant chemotherapy did not significantly improve event-free survival, while immune-related side effects more than doubled.
In the single-arm EPCORE NHL-2 cohort, adding epcoritamab to rituximab and lenalidomide produced responses in 95% of 41 untreated follicular lymphoma patients, with no control group to prove added benefit.
Dronabinol beat placebo on a nightmare-severity scale in 171 adults, but the effect was moderate and the only serious adverse events occurred in the drug group. Long-term safety is untested.
In a randomised trial of 3,102 bypass patients across seven countries, all-cause mortality was near-identical between bilateral and single internal thoracic artery grafting.
A 2,234-patient UK trial gave people with unexplained syncope a 14-day ECG monitor or usual care. A year on, fainting episodes were no fewer — a caution against confusing better detection with better outcomes.
Ervebo was licensed against Zaire ebolavirus. WHO says it is not known whether it protects against Bundibugyo virus in humans — which is why a trial is running alongside the wider rollout.
Lorecivivint improved pain modestly and changed nothing structural. Sprifermin grew cartilage and the effect made no difference by year five. Both illustrate the gap between a biomarker and a benefit.
A Federal Register notice opens a five-day comment window in September. The stated goal is to realign clinical operations across the institute and align with Presidential priorities.
TARGET-CTCA randomised 3,170 emergency patients whose troponin had cleared them of a heart attack. Outpatient CT angiography-guided care matched standard care on later infarction or cardiac death.
In 264 patients in China, the device maker's trial reported clinical worsening in 25.7% versus 51.5% on medical therapy alone (HR 0.49) — but no sham procedure blinded patients.
DANISH-CRT randomised 1,000 patients to electrically targeted or conventional left ventricular lead placement. After nearly four years, death and heart failure admissions were the same.
PRAGUE-26 randomised 558 patients across 11 Czech hospitals. The seven-day composite fell from 6.8% to 0.7%, driven by cardiorespiratory collapse, with no clear bleeding penalty.
PREMIUM tested prasugrel alone against aspirin plus prasugrel in 2,216 STEMI patients in Japan. Monotherapy failed to show noninferiority, and the confidence interval sat above one.
POET II randomised 508 stabilised patients to stop antibiotics or complete the standard course. The safety endpoint met noninferiority; relapse of infection did not go the same way.
LIBREXIA-ACS enrolled 14,194 patients before stopping for futility. Milvexian did not reduce events, but intracranial and fatal bleeding were identical to placebo, which is the class's whole premise.
CARDIO-TTRansform randomised 1,432 patients with transthyretin amyloid cardiomyopathy to eplontersen or placebo for 140 weeks. The confidence interval around the primary result crosses one.
Guidelines have hedged on anticoagulation for people with one stroke risk factor. SINGLE-AF randomised 1,803 patients in South Korea, and the events it counted were very few in both arms.
A meta-analysis of 90,000 people found these diabetes-origin drugs reduced kidney disease progression by 37% and cardiovascular events by 23%, and the benefit was similar in people who did not have diabetes.
The phase 3 VALOR trial met its primary endpoint at the higher dose of the oral TYK2-JAK1 inhibitor, with more serious infections — a benefit and a safety signal that arrive together.
ACACIA-HCM met both primary endpoints in symptomatic non-obstructive HCM. The gains were real but small, and one in ten patients had a reversible drop in pumping function.
A network meta-analysis found route of delivery decides everything, and that autologous transplant ranked below placebo. In IBS, 13 trials produced very low certainty of any effect.
A $12 million gift will test whether low-dose rapamycin changes the transition to frailty in adults over 65 — the first study of the drug designed around an outcome rather than a biomarker.
High urate levels track with worsening kidney disease, so treating them seemed a logical way to slow it. Two trials that lowered urate with allopurinol found the kidneys declined at the same rate anyway.
Five-year follow-up of a Chinese randomised trial shows paclitaxel–cisplatin–capecitabine before chemoradiotherapy lifted failure-free survival from 63% to 78% in high-risk disease — at the cost of more toxicity.
The longest human trial of low-dose rapamycin for healthy ageing found no change in visceral fat, its own primary outcome. The positive findings it is quoted for rest on eight women.
The MTA trial randomised 579 children and found medication management clearly superior short-term. Its own long-term follow-up found the treatment groups no longer differed by 6 to 8 years.
Post-bariatric hypoglycemia hits roughly 1 in 12 gastric bypass patients with sudden blood sugar crashes. Avexitide cut severe events by 55% against placebo.
A ten-person phase 1 study measured whether oral rapamycin reaches the central nervous system in early Alzheimer's. It did not — and several disease biomarkers rose over eight weeks.
Rezpegaldesleukin works by expanding the immune system's regulatory T cells rather than blocking an inflammatory signal — a new mechanism for atopic dermatitis, tested so far only in a phase 2b study.
In the phase 3 Launch-HTN trial, adults already on two to five drugs fell 9.1 mm Hg further on lorundrostat than placebo — the second aldosterone-synthase inhibitor to post positive data after baxdrostat.
ITACS randomised 955 anaemic adults to intravenous iron or placebo before elective cardiac surgery. Fewer patients needed a transfusion, but the primary benefit was a median of one day alive and at home.
A 100-child randomized trial in Uganda found a cheap supplement lowered infection rates on top of standard hydroxyurea therapy — though the authors say it needs a bigger trial before it changes practice.
ACT-South Asia randomised 1,847 patients in Nepal, Bangladesh and Pakistan. Treatment failed in 5.1% of each group — no benefit from the second antibiotic, and a point in favour of stewardship.
RACER-Knee masked patients with sham incisions and found a Forgotten Joint Score difference of −1.5 points at 12 months (P=0.62) — while the robotic operation cost more.
Adding an inhaled steroid to the as-needed inhaler roughly halved severe attacks against a short-acting reliever, at a fraction of the daily steroid exposure that maintenance therapy delivers.
VPM1002 did not show non-inferiority to BCG for preventing tuberculosis infection in a phase 3 trial across sub-Saharan Africa. Far fewer infections occurred than planned, leaving the comparison unresolved.
Observational studies link higher water intake to healthier kidneys. When a randomised trial coached people with kidney disease to drink more, their filtration fell at the same rate as the control group.
A phase 2 trial of dasatinib plus quercetin missed its bone endpoint. A senolytic injected into the eye produced a 5.6-letter vision gain whose confidence interval crosses zero.
A once-daily experimental pill beat placebo in a phase 3 trial and is under regulatory review — yet no study has tested its claimed edge over older dopamine agonists head-to-head.
Five-fraction SBRT improved several quality-of-life measures but was not superior to longer IMRT for prostate cancer. Three-year disease-free survival was 88.6% with SBRT and 92.1% with IMRT.
In a trial of 1,643 men, prostate cancer killed 3.1% of the monitored group and 2.2% of the surgery group — a difference the trial could not distinguish from chance. Metastases were twice as common.
In 2,414 children, the MODAM-MAM trial found one daily sachet of therapeutic food non-inferior to standard supplementary food; two sachets did marginally better but used far more product.
The radioligand cut the risk of radiographic progression or death by 28% when added to standard therapy in a phase 3 trial — a real but incremental gain, bought with more side-effects.
The first randomised trial to pit surgery against the natural course of big-toe arthritis found fusion cut walking pain to 1.3 on a 10-point scale at one year, versus 5.7 with watchful waiting.
Longevity trials want a measurement that moves in months instead of decades. A Berlin study ranked the candidates, and a four-week diet trial showed what a clock does when nothing much happens.
The label follows people for life, pushes them onto second-line antibiotics, and is associated with higher rates of MRSA and C. difficile. A randomised trial tested a faster way to remove it.
Analgesic trials keep failing, and one reason is that the comparison arm keeps improving. Where a trial recruits, how long it runs and how big it is all move the placebo response — and none of that is about the drug.
The randomised evidence in older men found a moderate effect on sexual function and none on vitality or walking. A 5,246-man safety trial cleared it on heart attacks and found more fractures.
Both things are true, and both rest on high-certainty evidence. The phase 3 trial and the meta-analysis that followed it measured different outcomes and reached opposite-sounding conclusions.
GLORY-2 randomised 461 Chinese adults with obesity to a weekly GLP-1/glucagon dual agonist or placebo; weight fell 16.65% versus 1.50% at 60 weeks, alongside high rates of nausea and vomiting.
Dazukibart eased skin and muscle disease within weeks and held up for nearly two years — but the trials were tiny, manufacturer-run, and the makers say a phase 3 is needed to confirm it.
A prespecified analysis of the ATTRibute-CM trial found the TTR-stabilising pill slowed the decline in how patients rated their own health, with a number-needed-to-treat of six to keep one 'not worse'.
In the phase 3 TORCH trial, following transarterial chemoembolisation with thermal ablation more than doubled median progression-free survival, to 17.7 months from 7.3, in unresectable liver-confined cancer.
The LEAP trial reversed a generation of avoidance advice. A second trial run in the general population missed its primary endpoint, and the distance between those two results is the part usually left out.
Patients in remission who stayed on the JAK inhibitor through a second year flared far less than those switched to placebo — 7.4% versus 59.5% — in an extension of the SELECT-GCA trial.
The final HARMONi-A analysis reports a 2.7-month gain in median overall survival — 16.8 versus 14.1 months — for the PD-1/VEGF bispecific added to chemotherapy in a China-only phase 3 trial.
In KEYNOTE-B15/EV-304, perioperative enfortumab vedotin plus pembrolizumab lifted two-year event-free survival to 79.4% from 66.2% against standard cisplatin-based chemotherapy in patients fit for it.
The MITRIC basket trial gave 12 patients with immunotherapy-resistant cancer stool transplants from patients who had responded. It was safe but produced no objective tumour responses.
The SMART trial tested clesrovimab against palivizumab in premature and heart- and lung-affected infants. It was built to show safety, not to prove one prevents more RSV than the other.
In the phase 3 CORALreef AddOn head-to-head, enlicitide dropped LDL cholesterol 64.6% at day 56 — far past ezetimibe, bempedoic acid, or the two combined.
A randomised-withdrawal trial showed the antibody-lowering drug kept CIDP patients in remission. A 2026 extension reports lasting benefit, but with no placebo group to measure it against.
The targeted combination more than tripled progression-free survival versus placebo in iodine-refractory disease — but overall survival is not yet significant and the trial was mostly Asian.
Petrelintide, a long-acting amylin analogue given without a GLP-1 drug, reduced bodyweight by up to 8.6% at 16 weeks across two small phase 1 trials and was well tolerated. The finding is early.
In the phase 3 BRUIN CLL-322 trial, adding pirtobrutinib to a fixed course of venetoclax and rituximab raised 24-month progression-free survival to 87% from 72% in previously treated disease.
In 328 patients with third-generation cephalosporin-resistant gut bacteria in the blood, an old, narrow-spectrum antibiotic met its non-inferiority mark, offering a way to spare carbapenems.
Years of observational data suggested 4CMenB cut gonorrhea risk by a third or more. A placebo-controlled trial in 654 people at high risk found essentially no effect at all.
The KADS extension followed 130 patients who had already been through a ketamine trial. Response fell from 30% at treatment's end to 17% a month later.
In the head-to-head OVERLORD-MS trial, 92.2% on rituximab and 94.8% on ocrelizumab had no new MRI lesions from months 6 to 24, meeting the prespecified bar for noninferiority.
An all-oral short regimen, its length set by chest-imaging severity, was non-inferior to 18-month treatment and caused fewer serious adverse events in a Chinese randomised trial.
In DigiSep, adding metagenomic sequencing to standard cultures did not improve the main 28-day outcome, though ventilation time and shock resolution were shorter in exploratory analyses.
Oral paltusotine kept median IGF-I near the normal ceiling through week 207 in an open-label extension, but only 20 of 43 patients remained by then and the study had no comparison group.
A sham-controlled trial found FMT shifted bacterial composition but cleared drug-resistant organisms no better than a saline placebo. A second trial shows what a defined replacement product can and cannot yet prove.
REMEASURE randomised 73 patients to Kono-S anastomosis or mesenteric resection after ileocolic surgery for Crohn's disease; endoscopic recurrence at six months was 33% versus 32%.
In ATTRACTION, tirofiban given after successful thrombectomy left more stroke patients independent at 90 days than placebo, though symptomatic brain bleeds trended higher and were not significantly different.
In a 500-patient ICU trial across seven countries, bicarbonate infusions for metabolic acidosis in patients on vasopressors did not lower major kidney events or death at 30 days.
Final ALIGN results show the endothelin blocker preserved kidney function over 2.5 years in a rare kidney disease, yet the difference at week 136 just missed statistical significance (p=0.057).
In a UK trial of 1,956 critically ill adults, neither carbocisteine nor nebulised hypertonic saline shortened time on a ventilator, and each was tied to a specific harm.
In two industry-funded phase 3 studies, every child followed at least 16 months met the goal — but all had a grade 3 or 4 event, and one died of a conditioning-drug complication.
In the FIND-CKD trial, non-diabetic patients on finerenone lost kidney function at 3.3 versus 4.0 ml/min/1.73 m² a year — a small edge that extends a diabetes drug to a new group.
In a 122-patient South African proof-of-concept study, a four-month all-oral combination of quabodepistat, delamanid and bedaquiline cleared TB from sputum as often as the six-month standard.
In the phase 3 GCAptAIN trial the interleukin-17A blocker did not significantly beat placebo for sustained remission at one year, leaving the IL-6 inhibitor tocilizumab as the proven steroid-sparing option.
In the MajesTEC-9 trial, the bispecific antibody teclistamab cut the risk of progression or death by 71% against standard three- and two-drug regimens. Serious infections were common.
The anti-alpha-synuclein antibody did not significantly delay motor progression in the phase 2b PADOVA study, though a non-significant trend and secondary signals keep a phase 3 trial alive.
Two GSK-funded phase 3 trials found roughly 20% of patients on bepirovirsen reached a functional cure at 72 weeks versus none on placebo — alongside more adverse events and liver-enzyme rises.
In the phase 3 LIBRETTO-432 trial, adjuvant selpercatinib raised two-year event-free survival to 92% from 61% in resected stage II-IIIA RET fusion-positive lung cancer. The trial was small and survival is unproven.
35% of patients on tezepelumab stopped oral steroids entirely, versus 21% on placebo, without losing asthma control — in a drug that targets inflammation upstream of the eosinophil count doctors usually check.
In the phase 3 DRAGON-01 trial, delivering paclitaxel directly into the abdominal cavity on top of standard chemotherapy raised median survival to 19.4 months from 13.9 in gastric cancer with peritoneal spread.
A new structured dataset finally makes the approvals countable. Two trials published weeks apart show why counting them says little: 0.86 fewer migraine days in one, a 20-point symptom shift in the other.
Two large trials of the anti-ST2 antibody gave discordant results: a modest signal in the phase 2b study, and a missed primary endpoint for the same dose in the confirmatory phase 3.
In the phase 3 Integral-1 trial, cefepime plus the new β-lactamase blocker nacubactam cured 82% of complicated urinary infections versus 61% for imipenem, offering a way to spare carbapenems.
EPISODE randomized 144 people with treatment-resistant depression across four arms. High-dose psilocybin missed its main outcome, even as secondary measures showed symptom benefit.
A study running 5.3 million evaluations through nine large language models found eligibility judgments were largely stable across identity labels — except when a patient vignette mentioned homelessness.
In VERITAC-2 the oral degrader more than doubled progression-free survival over fulvestrant in patients with an ESR1 mutation — 5.0 months versus 2.1 — but not in the trial as a whole.
In 35 patients with recurrent major depression, psilocybin beat an active placebo through day 42. On self-reported measures the effect lingered further, but the trial's primary endpoint showed no difference at 365 days.
TSND-201 improved PTSD severity, disability, and depression scores over placebo in 65 participants — delivered as an oral dose with monitoring but no formal psychotherapy alongside it.
Buntanetap showed dose-dependent cognitive benefit in biomarker-positive patients with mild disease. The company's announcement does not say what happened in the full 351-patient population.
PRoMPT BOLUS, a 9041-child trial across 47 emergency departments, found kidney-injury events at 30 days in 3.4% on balanced fluid and 3.0% on saline (P=0.85) — no meaningful difference.
Cemdisiran, a gene-silencing drug given under the skin once every three months, cut daily-living scores more than placebo in antibody-positive generalised myasthenia gravis, a rare complement-driven disease.
Most hiccups stop on their own, and the remedies everyone swears by have almost no trial evidence. The one gadget with published data was studied by its own inventor and funded through the product crowdfunding campaign.
A systematic review of 27 studies finds women reach higher drug concentrations at the same psychotropic dose. A separate review of 103 atrial fibrillation trials finds 43% under-enrolled women against disease prevalence.
Two doses of the long-acting antibody L9LS protected under-fives in a high-transmission area, but researchers say a higher dose is likely needed for stronger cover.
In an industry-funded, open-label trial of 3,048 patients with heart disease, targeting LDL below 55 rather than 70 mg/dL lowered a composite of cardiovascular events over three years.
Results posted to ClinicalTrials.gov show 15- and 25-day nirmatrelvir-ritonavir regimens produced no meaningful edge over placebo on cognitive, autonomic, or exercise-intolerance symptoms.
In 1,362 Shanghai dialysis patients followed for over three years, hemoadsorption plus haemodialysis lowered all-cause mortality versus dialysis alone — a borderline result from an open-label trial.
Relacorilant kept blood pressure under control when patients who had responded were kept on it rather than switched to placebo. The pivotal trial randomised just 62 people.
In the phase 3 ATOMIC trial, atezolizumab on top of standard FOLFOX chemotherapy raised three-year disease-free survival to 86.3% from 76.2% after surgery for stage III dMMR colon cancer.
In the phase 3 PATCH trial, oestrogen skin patches were non-inferior to LHRH injections for three-year metastasis-free survival, with fewer hot flushes but more breast swelling.
In a single-centre trial of 100 patients with decompensated cirrhosis, six months of atorvastatin cut recurrent complications from 72% to 36% — a striking but preliminary result needing larger confirmation.
Up to eight sessions of psilocybin, layered onto a small randomized comparison against an active placebo, produced a 73% response rate by the end of treatment. The trial was designed to test safety first.
OCULUS was halted at interim analysis: continuing a GLP-1 or GIP agonist before endoscopy left 25.0% of patients with clinically significant retained stomach contents, versus 3.1% who held one dose.
In a Chinese phase 2–3 trial, neoadjuvant chemotherapy, lenvatinib and a PD-1 antibody before liver surgery lifted median event-free survival to 18.0 months from 8.7. The survival signal was not yet conclusive.
In the open-label extension of the VIVID programme, remission rates stayed high at 104 weeks — but no placebo group ran alongside, so durability is described, not proven, against nature.
In the COSPIRIT-JIA trial, most children with two stubborn forms of juvenile idiopathic arthritis responded to ixekizumab by week 16 — but the main result had no control group.
In the ALLEGORY trial, 76.7% of patients with active SLE reached the SRI-4 response at 52 weeks on the B-cell–depleting antibody versus 53.5% on placebo, extending a drug already cleared for lupus nephritis.
Bimagrumab targets fat, not appetite. Paired with semaglutide in 507 adults with obesity, the combination out-performed either drug alone at 48 weeks — with its own separate set of side effects.
A three-year update of RATIONALE-309 found tislelizumab plus chemotherapy lengthened median progression-free survival to 9.6 months from 7.4. The overall-survival gain was not statistically significant on its own.
reSET, Somryst and EndeavorRx cleared a real regulatory bar, on real trial data. The pivotal ADHD game moved an attention score by 0.88 of a point. Clearance is not the obstacle the category keeps hitting — payment is.
In KEYNOTE-905/EV-303, adding enfortumab vedotin and pembrolizumab to surgery lifted two-year event-free survival to 74.7% from 39.4%. Serious side effects were common.
In the LATITUDE trial, monthly cabotegravir–rilpivirine injections cut regimen failure to 22.8% from 41.2% on oral therapy among people with a history of missed doses, prompting an early stop.
Efimosfermin, an FGF21 analogue given every four weeks, was well tolerated over 24 weeks in 84 patients with fatty-liver disease — but this trial was designed to test safety, not whether it heals the liver.
A trial across five Chinese hospitals found remote robotic urological surgery non-inferior to local surgery. The sample is small, the surgeons were experts, and the network never failed.
In the phase 3 ASCENT-04 trial, sacituzumab govitecan with pembrolizumab lifted median progression-free survival to 11.2 months from 7.8 in PD-L1-positive disease. Survival data are immature.
In the RECOVER extension, about a fifth of participants were in remission at 24 months and some who had not responded at 12 months did later. The second year had no control group.
FDA approved copper histidinate on the strength of two single-arm trials compared with an external control group. Children treated within four weeks of birth had a 78 percent lower risk of death.
Standard questionnaires found no daytime difference between suvorexant and placebo. Four-times-daily smartphone prompts found fatigue worse in the morning and better later. The trial had 40 people.
The biomarker effect reported at CTAD is large and sustained over two years. The sample is seven, the design is open dose-escalation, and imaging was available for three.
TRIUMPH-4 tested retatrutide in 445 adults with obesity and knee osteoarthritis. The weight numbers are striking. So are the discontinuation numbers sitting beside them.
Bristol Myers Squibb found 'irregularities' in how a small number of ADEPT-2 sites executed the study, excluded their patients, and is enrolling more. Alzheimer's-related psychosis still has no approved treatment.
Semaglutide moved Alzheimer's biomarkers without moving the clinic. A separate liraglutide trial missed its primary endpoint on brain metabolism. Together they close a decade-old hypothesis — or at least narrow it.
Two November trials found ambient AI cut documentation time and work exhaustion. A parallel emergency department comparison found physicians spent more time in the note, not less.
In ATTAIN-2, the highest dose of the oral GLP-1 pill cut body weight 9.6% over 72 weeks in adults with type 2 diabetes, against 11.2% in the earlier trial of adults without diabetes.
A TB vaccine financing plan, six target product profiles for children's cancer drugs, and a paediatric research agenda. All three are attempts to specify a product before a market decides to build it.
SURPASS-CN-INS randomised 257 adults at 26 Chinese hospitals. The 10 mg and 15 mg doses performed almost identically against placebo, and diarrhoea affected roughly a third of those on the higher doses.
Seltorexant held its effect over two weeks while zolpidem's faded, in a 364-person study run in 2017-2019 and published only now. The delay is disclosed in the paper itself.