AstraZeneca's eneboparatide meets the Phase 3 CALYPSO primary endpoint in chronic hypoparathyroidism with full data presented at the European Congress of Endocrinology, Alkermes' LUMRYZ meets its primary endpoint in the Phase 3 REVITALYZ trial for idiopathic...
Alzheimer’s disease (AD) represents a significant medical concern for all of us as we grow older, as the NIH has published that the average risk of dementia over 55 years old is now 42%. AD is presently affecting 6.9 million Americans aged 65 or older and is expected...
Cytokinetics’ aficamten meets dual primary endpoints in the pivotal Phase 3 ACACIA-HCM trial for non-obstructive hypertrophic cardiomyopathy, Axsome Therapeutics secures FDA approval of AUVELITY as the first non-antipsychotic treatment for Alzheimer’s disease...
Children have long been described as therapeutic orphans. It is a term first used by the American paediatrician Harry Shirkey of the Children’s Hospital in Birmingham, Alabama, at a 1963 conference and formally published in 1968. Despite more than five decades of...
For decades, clinical trials have failed to adequately represent the patients who will ultimately use the medicines and treatments being tested. Gaps in enrolment across race, ethnicity, sex, age, and socioeconomic background have created an incomplete evidence base,...
Etavopivat delivers Phase 3 success in sickle cell disease, satralizumab becomes the first positive Phase 3 result in MOGAD, AstraZeneca completes its triple-positive COPD biologic programme, and a busy week for paediatric medicine brings new approvals and data in...
Dr Steven Cox, CEO of the research charity, Cardiac Risk in the Young (CRY) explains the key findings and importance of a seminal study (reviewing cardiac screening data collated over a 10 year period), published on 24 February 2026 in the Journal of the American...
Couples trying to conceive are bombarded with supplement recommendations. Scroll through any fertility forum and you'll find confident claims about CoQ10, vitamin D, and a dozen other compounds that supposedly hold the key to conception. Some of these recommendations...
Wouldn’t life be easier if someone was there to keep on top of chores around the house? That’s the idea behind Anavex Life Sciences’ experimental, once-daily pill blarcamesine, which is being tested in patients in the early stages of Alzheimer’s disease. This drug...
The next operational lessons for early-stage clinical trials in humans might not come from pharma but from a cattle ranch in Nebraska. While human clinical research debates decentralized trials, adaptive protocols, novel technologies, and new approaches to oversight,...
New research suggests that inflammatory and microstructural changes in a key brain reward region may differ between chronic depression and acute depressive symptoms, offering fresh insight into the biological mechanisms underlying the condition. In a large scale...
The Functional Gut Clinic (FGC), the UK’s leading provider of specialist gastrointestinal (GI) diagnostics, today announced the launch of its dedicated GI Clinical Trials establishing the organisation as the country’s first boutique, end‑to‑end clinical research...
Pharvaris Announces Positive Phase 3 Results for Deucrictibant as On Demand HAE Treatment Pharvaris has announced positive topline data from its global Phase 3 RAPIDe 3 study, confirming that the oral bradykinin B2 receptor antagonist deucrictibant shows strong...
Otsuka Gains Approval for First of Its Kind Treatment Against Rare Kidney Disease In a significant milestone for kidney disease care, Otsuka Pharmaceutical has received accelerated approval from the US Food and Drug Administration for a new therapy for Immunoglobulin...
Engasertib Trial Offers New Hope for Patients with Hereditary Hemorrhagic Telangiectasia A recent clinical study has delivered encouraging results for people living with hereditary hemorrhagic telangiectasia, a rare genetic vascular disorder that causes chronic and...
Pharmaceutical approaches to ALS remain sparse and "hard-to-develop". Riluzole (approved 1995) and edaravone (IV in 2017, oral in 2022) only offer modest benefits. More recently, Biogen's Qalsody (tofersen) was approved for SOD1-mutant ALS. But a new wave of new...
Miraculous Progress for 3-Year-Old After Ground-Breaking Gene Therapy for Hunter Syndrome A three-year-old boy, Oliver Chu, has astounded doctors after becoming the first person in the world with Hunter syndrome also called MPS II to receive a pioneering gene therapy....
Clinical trials have long been the backbone of medical innovation, providing the evidence base for new treatments, diagnostics, and preventive measures. Yet for decades, the traditional model, site-based, paper-driven, and geographically limited, has been plagued by...