Preclinical and observational studies suggest that exercise may improve cancer outcomes. However, definitive level 1 evidence ...

Chimeric antigen receptor (CAR) T cells targeting CD19 have transformed the treatment of B-cell cancers, but many patients do not have long-term remission. We designed an anti-CD19 enhanced ...

Narcolepsy type 1 is a disorder of hypersomnolence caused by a loss of orexin neurons, which results in low orexin levels in the brain. In this phase 2, randomized, placebo-controlled trial ...

In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder. 2 Department of Chemistry and Biochemistry, University of ...

Musunuru and colleagues 1 describe in the Journal an innovative approach in which gene editing is applied in the treatment of a very rare disease, the severe urea-cycle disorder carbamoyl ...

In this open-label trial with a 2-by-4 factorial design, we randomly assigned children with HIV who had first-line treatment failure to receive second-line therapy with tenofovir alafenamide ...

In the past 25 years — from 2000 to 2024 — we have witnessed a revolution in the management, treatment, and outcome of what used to be the most lethal hematologic cancer: Philadelphia ...

A total of 2516 participants underwent randomization; 1797 (71.4%) completed the trial. Of 2421 participants in the full analysis population (1209 assigned to the albuterol–budesonide group and ...

Each year, millions of pulmonary nodules are identified incidentally or through lung cancer screening, and many involve biopsy to distinguish cancer from benign processes. Both navigational ...

Patients with narcolepsy type 1 have hypersomnolence caused by a loss of orexin-producing neurons. New research findings on oveporexton, a highly selective oral orexin receptor 2 agonist, in these ...

Nerandomilast (BI 1015550) is an orally administered preferential inhibitor of phosphodiesterase 4B with antifibrotic and immunomodulatory properties. Nerandomilast has been shown to slow the ...

New AI-based software for generating patient notes purposely filters out social “chitchat,” but a primary care physician sees serious risks in that practice, for doctors and patients alike.