Archive
1250 articles, sorted by publication time.
An exploratory analysis of CheckMate 77T shows that ctDNA clearance and pathological complete response may complement each other in identifying prognosis; machine learning integrates and ranks multiple clues, but prospective validation is still lacking before treatment can be intensified or reduced on this basis.
After videos of Chinese patients sparked a wave of interest, the United States and Singapore have begun evaluating cervical lymphatic-bypass surgery in regulated studies; the preliminary signals are striking, but small samples, a lack of controls, and an unclear mechanism are still insufficient to prove that it can reverse dementia.
Sangamo’s single-dose AAV therapy has already had portions of its U.S. marketing application submitted; PTC is betting not only on early clinical signals, but also on whether the manufacturing documentation can be completed in the fourth quarter and clear the most challenging regulatory hurdle for gene therapies.
An interim analysis of REZILIENT3 showed that the targeted therapy combined with platinum-based chemotherapy can prolong progression-free survival; however, the magnitude of benefit, overall survival, and safety data have not yet been disclosed, and whether the regimen can reshape treatment choices awaits scrutiny of the full results.
The Duchenne cell therapy deramiocel preserved some upper-limb function in a Phase 3 trial but failed to convince experts that evidence of efficacy against cardiomyopathy was sufficient; a statistical correction and manufacturing inspection add further uncertainty to the final decision.
The once-daily pyruvate kinase activator showed some hematologic activity in a Phase 2 trial, but lacked a clear dose response and failed to clear the increasingly competitive bar set by drugs in the same class.
Canada has authorized the first-in-human trial, and the first participants have already been vaccinated. For a virus type with no dedicated vaccine or treatment, the study will test whether the mRNA platform can translate rapid development into a usable immune response.
RSM01 is set to be evaluated for the first time in infants aged two weeks to eight months for safety and pharmacokinetics; this small, early-stage trial will not answer questions about protective efficacy, but it is a critical gateway to later-stage development.
This regulatory designation opens a channel for more frequent FDA communication on Phanes’ dual-targeting CLDN18.2/CD47 strategy, but its actual clinical value must still be demonstrated by the ongoing early-stage trial, which is currently recruiting.
The oral antiviral will undergo randomized controlled testing in infants and young children hospitalized with severe disease in the United States. Phase 3 results from China provide a signal of efficacy, but regional differences, resistance mutations, and the limited number of severe cases remain questions that must be answered in multinational development.
Intracellular polyamines do more than support growth and protein synthesis: they also constrain reactive iron that can readily cause oxidative damage. The finding offers a new hypothesis for combination cancer therapy and may also connect metabolic clues in some forms of early-onset Parkinson’s disease.
A small randomized trial suggests that pharmacologic-dose intravenous vitamin C combined with chemotherapy may prolong survival in patients with metastatic pancreatic cancer; however, this does not vindicate oral supplements and is not yet sufficient to rewrite treatment standards.