In two Phase 3 trials, about 19–20% of selected participants reached the study-defined functional-cure endpoint; most did not, and functional cure is not sterilising eradication of HBV.
Adults with obesity or overweight and type 2 diabetes receiving the investigational 12 mg retatrutide dose lost an average 20.8% of body weight over 80 weeks in TRIUMPH-2.
The small-molecule GLP-1 receptor agonist safiglipron improved glucose control and reduced weight in the Phase 3 OUTSTAND-1 trial, without specific fasting or meal-timing restrictions.
The FDA has approved zilurgisertib for adults and children aged 12 years and older with fibrodysplasia ossificans progressiva, an ultra-rare genetic disease that causes abnormal bone to form in muscles and other connective tissues. The oral drug reduced new abnormal bone formation in a small randomized trial, but it does not reverse bone that has already formed or prove that established disability can be restored.
The FDA has approved belzutifan plus lenvatinib for adults with advanced clear-cell kidney cancer after previous PD-1 or PD-L1 immunotherapy. In a randomized trial of 747 patients, the combination delayed cancer progression and produced more tumour responses than cabozantinib, although a statistically significant overall-survival benefit was not demonstrated.
The FDA has approved obinutuzumab for patients aged 2 years and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome who are already in remission. In the randomized Phase 3 INShore trial, the B-cell-targeted treatment maintained remission more effectively than mycophenolate mofetil, but it also carries important immune-suppression risks.
A large multicentre study published in Nature Medicine found that an AI system could identify many esophageal cancers — including some early cancers — on non-contrast chest CT scans. The technology could potentially extract additional cancer-screening information from scans already being performed, but it does not replace endoscopy and has not yet been shown to reduce cancer deaths.
The investigational treatment ulefnersen has met the primary endpoint of the Phase 3 FUSION trial in people with ALS caused by FUS gene mutations. The RNA-targeted therapy is designed to reduce production of FUS protein, but detailed Phase 3 results have not yet been peer-reviewed and the treatment is not yet approved.
A randomized NHS trial involving more than 142,000 people found that adding the Galleri multi-cancer blood test to usual care did not significantly reduce the trial's primary measure of stage III and IV cancers. Some secondary findings were more encouraging, including fewer stage IV cancers, but the evidence does not yet show that the test reduces cancer deaths or should replace established screening programmes.
The FDA has approved imlunestrant with abemaciclib for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after progression on endocrine therapy. The decision adds another precision-treatment option guided by a tumour mutation that can be detected in circulating tumour DNA.
The FDA has approved levacetylleucine for ataxia associated with ataxia-telangiectasia, creating the first approved treatment specifically for this neurological feature of the rare inherited disease. A randomized Phase 3 trial found improved ataxia scores over 12 weeks, but whether the treatment changes long-term neurodegeneration remains unknown.
The FDA has approved rebisufligene etisparvovec-hopf, a one-time gene therapy for eligible children with Sanfilippo syndrome type A, marking the first approved treatment for this rare inherited neurodegenerative disease. The evidence suggests better preservation of cognitive development than seen in an untreated natural-history group, but the pivotal comparison was not a conventional randomized placebo-controlled trial.
The FDA has approved rebisufligene etisparvovec-hopf, a one-time gene therapy for eligible children with Sanfilippo syndrome type A, marking the first approved treatment for this rare inherited neurodegenerative disease. The evidence suggests better preservation of cognitive development than seen in an untreated natural-history group, but the pivotal comparison was not a conventional randomized placebo-controlled trial.
A first-in-disease Phase 1 trial found deep B-cell depletion and encouraging remission signals after treatment with the experimental bispecific antibody A-319 in people with active lupus. The approach could eventually offer a simpler alternative to individualized CAR-T therapy, but the study involved only 12 patients and cannot yet establish effectiveness.
An experimental inhaled RNA medicine successfully reduced a selected inflammatory protein inside the lungs in an early human trial, suggesting that gene-silencing medicines may eventually be delivered directly to lung tissue.
A genetically modified pig kidney worked for 271 days in a man with kidney failure, allowing him to avoid dialysis before he later received a human donor kidney.
U.S. regulators approved Casgevy in December 2023 — the world's first medicine based on CRISPR-Cas9 gene editing — offering a potential one-time functional cure for sickle cell disease and marking a watershed moment in the history of genetic medicine.
Insulin efsitora alfa-gobe (Onswik) is the second US-approved once-weekly basal insulin for adults with type 2 diabetes.
The FDA has approved obinutuzumab for patients aged 2 years and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome who are already in remission. In the randomized Phase 3 INShore trial, the B-cell-targeted treatment maintained remission more effectively than mycophenolate mofetil, but it also carries important immune-suppression risks.
The FDA has approved belzutifan plus lenvatinib for adults with advanced clear-cell kidney cancer after previous PD-1 or PD-L1 immunotherapy. In a randomized trial of 747 patients, the combination delayed cancer progression and produced more tumour responses than cabozantinib, although a statistically significant overall-survival benefit was not demonstrated.
The FDA has approved zilurgisertib for adults and children aged 12 years and older with fibrodysplasia ossificans progressiva, an ultra-rare genetic disease that causes abnormal bone to form in muscles and other connective tissues. The oral drug reduced new abnormal bone formation in a small randomized trial, but it does not reverse bone that has already formed or prove that established disability can be restored.
Mpox (වඳුරු උණ) වෛරසයේ ව්යාප්තිය පිළිබඳව ශ්රී ලංකා සෞඛ්ය අංශ සහ ලෝක සෞඛ්ය සංවිධානය (WHO) දැඩි අවධානයෙන් පසුවේ. අප්රේල් 13 වන දින වන විට, මෙරට තුළ වෛරසය පැතිරීමේ ක්ෂණික තර්ජනයක් නොමැති වුවද, ජාත්යන්තර සංචාරකයින් හරහා සිදුවිය හැකි ආසාදනය වීම් වළක්වා ගැනීමට පියවර ගෙන ඇත.
ස්ථූලතාවයට එරෙහිව සටන් කළ හැකි නවතම විද්යාත්මක සොයාගැනීමක් ලෙස, මොළයේ ඇති 'Astrocytes' නම් තාරකා හැඩැති සෛල මගින් ආහාර රුචිය පාලනය කරන ආකාරය පර්යේෂකයන් විසින් සොයාගෙන ඇත. මෙය ඉදිරියේදී අතුරු ආබාධවලින් තොර බර අඩු කිරීමේ ඖෂධ නිපදවීමට විශාල පිටුවහලක් වනු ඇතැයි අපේක්ෂා කෙරේ.
The World Health Organization declared mpox a public health emergency of international concern in August 2024 — its second such declaration in two years — as a new, more transmissible clade Ib strain spread rapidly through the Democratic Republic of Congo and neighbouring countries.
