Preclinical and observational studies suggest that exercise may improve cancer outcomes. However, definitive level 1 evidence is lacking. In this phase 3, randomized trial conducted at 55 centers, ...

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 ...

Yearly Covid-19 Booster Recommendations in Canada, Europe, and Australia. Although the rapid development of multiple Covid-19 vaccines in 2020 represents a major scientific, medical, 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 ...

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.

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 ...

Musunuru and colleagues 1 describe in the Journal an innovative approach in which gene editing is applied in the treatment of a very rare disease, the severe urea-cycle disorder carbamoyl ...

Chimeric antigen receptor (CAR) T cells targeting CD19 have transformed the treatment of B-cell cancers, but many patients do not have long-term remission. We designed an anti-CD19 enhanced ...

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 ...

Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival of 75 to 80%.