We treated 428 patients who had active rheumatoid arthritis despite methotrexate therapy with placebo or infliximab, a chimeric monoclonal antibody against TNF-α, in intravenous doses of 3 or 10 ...

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 ...

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

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 ...

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 ...

Medical House Pupil, Massachusetts General Hospital, 1901; Assistant in Pediatrics, Harvard University; Assistant Physician at the Children's Hospital; Assistant Physician and Pathologist at the ...

Next-generation sequencing has enabled precision therapeutic approaches that have improved the lives of children with rare diseases. Congenital diarrhea and enteropathies (CODEs) are associated ...

We and others identified retinoblastoma features that suggested that the tumors originate in long- and medium-wavelength cone precursors, and we discovered features of these cone precursors that ...

This review article presents two true-life clinical vignettes that illustrate how digital health technology can aid providers caring for patients with epilepsy. Specific information that would ...

Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit ...

Hormone absence or inactivity is common in congenital disease, but hormone antagonism remains controversial. Here, we characterize two novel homozygous leptin variants that yielded antagonistic ...

The specialty of critical care has achieved important advances in survival for many patients with the most complex disorders. Along with these advances, however, there has been an increasing ...