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598 articles, sorted by publication time.
The 30-person trial will assess whether POLB 001 can prevent cytokine release syndrome, which is common during treatment with the bispecific antibody teclistamab; if results are positive, it could change monitoring models for some blood cancer immunotherapies, but it remains early-stage clinical validation for now.
The U.S. FDA has approved teplizumab for children and adolescents aged 8 to 17 with recently diagnosed stage 3 type 1 diabetes to delay the decline in insulin secretion capacity; the decision is an accelerated approval, and clinical benefit still needs to be verified in postmarketing studies.
After the U.S. FDA allowed expanded access to daraxonrasib, Phase 3 survival data at the ASCO meeting explained why this RAS inhibitor is drawing intense attention; however, it is still not a formally approved therapy, and its efficacy, safety, and appropriate patient population remain subject to full review.
The U.S. FDA has agreed to allow Revolution Medicines to initiate an expanded access treatment protocol for daraxonrasib, targeting previously treated patients with metastatic pancreatic ductal adenocarcinoma; this is not marketing approval, and efficacy and safety still require further review and confirmation.
Regeneron’s Otarmeni has received FDA accelerated approval for certain patients with OTOF-related severe to profound sensorineural hearing loss; early trial data showed hearing improvement in most participants, but continued approval still requires further verification of clinical benefit.
A UCLH and UCL team observed in an NHS trial in the UK that 5 patients with severe lupus entered remission after receiving genetically modified T-cell therapy; the findings remain early, and whether it can become a routine treatment depends on larger, longer-term studies.
A validation study by a team involving the Broad Institute showed that noninvasive fetal sequencing produced results close to those from amniocentesis and chorionic villus sampling for most clinically relevant variants; but if it moves toward broad screening, it will still have to confront variants of uncertain significance, counseling burdens, and policy boundaries.
The U.S. National Institutes of Health has newly established ORIVA, which will coordinate the development, validation, and regulatory translation of new methodologies, with the goal of reducing animal use in appropriate contexts; however, its actual impact will still depend on subsequent funding, standards, and regulatory adoption.
lonvoguran ziclumeran reduced attack rates versus placebo in an international, double-blind phase 3 trial; the study has been published in NEJM, but long-term efficacy, rare risks, and regulatory review still require further confirmation.
A QIMR Berghofer team analyzed data from more than 85,000 participants of European ancestry, linking mole formation to immune regulation and cell proliferation pathways; the research still needs validation in more populations and clinical settings.