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 ...

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 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 ...

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 ...