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 ...

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 ...

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 ...

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.

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 ...

There are currently no approved treatments for nonrelapsing secondary progressive multiple sclerosis. New research findings examining the efficacy and safety of the BTK inhibitor tolebrutinib are ...

A 68-year-old woman presented to the emergency department with dizziness and shortness of breath with exertion.… Each interactive case presents an evolving patient history and a series of ...

Tolebrutinib is a BTK inhibitor that is being studied for the treatment of relapsing multiple sclerosis. New research findings comparing tolebrutinib with teriflunomide are summarized in a short ...

The past two decades have brought revolutionary changes in global health, driven by popular concern over the acquired immunodeficiency syndrome (AIDS), new strains of influenza, and maternal ...

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 ...

Shown are the genetic variants identified in EPO, the gene encoding erythropoietin (EPO), in patients with erythrocytosis. The term cDNA denotes complementary DNA, F female, M male, and NA not ...