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1250 articles, sorted by publication time.

New Drugs and Regulation · global

GSK’s First Lung Cancer Drug Wins FDA Approval: Jideytro Enters Later-Line ROS1-Targeted Treatment Market

For patients with advanced non-small cell lung cancer whose disease continued to worsen after treatment with a ROS1 inhibitor, Jideytro shrank tumors in 44% of patients in a single-arm trial. This approval, completed ahead of schedule, fills a later-line treatment need, but comparative efficacy and long-term safety still require further evidence.

Precision Medicine · us

Mutation-Tailored Antisense Drugs Reduce Seizures in Two Boys With Severe Epilepsy

Two single-patient trials used personalized antisense oligonucleotides to selectively suppress disease-causing SCN2A gene transcripts. After more than two years of treatment, both boys showed improvements in seizures and developmental abilities, but whether the benefits will persist and whether safety will withstand longer-term scrutiny remain to be determined through follow-up.

Clinical Trials · global

Phase 3 Intravesical Immunotherapy Trial: Tumors Completely Disappeared in Three-Quarters of Patients, With an 81% Bladder-Preservation Rate at Two Years

Cretostimogene produced durable responses in patients with high-risk bladder cancer after BCG treatment failure, with no treatment-related adverse events of grade 3 or higher; however, the study had no control group, so it cannot yet establish whether the therapy is superior to other bladder-preserving treatments.