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 ...

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 ...

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 ...

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

Multiple sclerosis is the most common cause of progressive neurologic disability in young adults. There had been no treatments for the disease, except for glucocorticoids, which were used for more ...

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 ...

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 ...

Sarcoidosis, a granulomatous disease that affects the lungs and other organs, has a variable course. Although spontaneous resolution of sarcoidosis occurs in some patients, half the patients with ...

Nerandomilast (BI 1015550) is an orally administered preferential inhibitor of phosphodiesterase 4B with antifibrotic and immunomodulatory effects. In a phase 2 trial involving patients with ...

A 29-year-old woman was admitted to the hospital because of sore throat and peritonsillar swelling and bleeding. Symptoms did not abate with antimicrobial therapy. A diagnosis was made. This case ...

The International Agency for Research on Cancer (IARC) classified alcoholic beverages as carcinogenic to humans (Group 1) on the basis of sufficient evidence of causality for seven types of cancer ...

These epidemiologic patterns highlight the limitations of focusing on genetics alone. Although the Human Genome Project greatly expanded our understanding of hereditary contributions to disease ...