1. In this phase 1–2 open-label study, autologous lentiviral hematopoietic stem-cell gene therapy for cystinosis was associated with sustained engraftment...

1. In this randomized controlled trial, once-daily oral enlicitide was associated with sustained reductions in low-density lipoprotein cholesterol levels compared...

1. Among patients with previously untreated programmed death-ligand 1 positive, triple-negative breast cancer, first-line sacituzumab govitecan plus pembrolizumab significantly prolonged...

1. In this open-label phase 1–2 study, pediatric patients with mucopolysaccharidosis type II treated with tividenofusp alfa showed substantial reductions...

1. In a large randomized trial of adolescent girls, one dose of either the bivalent or nonavalent human papillomavirus (HPV)...

1. A nucleoside-modified mRNA (modRNA) influenza vaccine showed greater protection against influenza-like illness compared to a standard inactivated vaccine in...

1. In this randomized trial, aficamten monotherapy led to greater improvements in exercise capacity compared to metoprolol in patients with...

1. In this large global cohort study, individuals aged 50 without five major cardiovascular risk factors (hypertension, hyperlipidemia, abnormal body...

1. In patients with ER-positive, HER2-negative advanced breast cancer who developed an ESR1 mutation during first-line aromatase inhibitor plus CDK4/6...

1. In this prospective cohort study, a single intravenous dose of scAAV2/8-LP1-hFIXco gene therapy in men with severe hemophilia B...