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

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 ...

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 ...

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 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 ...

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 ...

A total of 14 million Medicare beneficiaries receive the Low-Income Subsidy (LIS), which reduces cost sharing in Medicare Part D. Losing the LIS may impede medication access and affect mortality.

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 phase 2 trials involving patients with stage 2 or 3 fibrosis caused by metabolic dysfunction–associated steatohepatitis (MASH), efruxifermin, a bivalent fibroblast growth factor 21 (FGF21 ...

Tirzepatide and semaglutide are highly effective medications for obesity management. The efficacy and safety of tirzepatide as compared with semaglutide in adults with obesity but without type 2 ...

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 ...

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.