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 ...

We thank the patient and his family for their participation in this study; all the persons involved in the rapid manufacturing and evaluation of k-abe, including Kamila Wlodarczyk, Nicholas Tougas ...

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 ...

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 ...

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 ...

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 ...

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 ...

Smoldering multiple myeloma is an asymptomatic precursor of active multiple myeloma. Daratumumab monotherapy may help prevent progression to active myeloma in patients with smoldering disease.

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 ...

Dr. Maxwell Green (Medicine): A 70-year-old man presented to the emergency department of this hospital because of a 6-week history of anorexia, weakness, and weight loss. Six weeks before this ...