A 4-year-old boy presented with fatigue and frequent falls. After falling, he could not stand up using his legs and would instead push himself up with his hands. The aminotransferase levels were ...

A new European Union regulation introduces a centralized clinical assessment process for comparing the effectiveness and safety of new medicines with those of the standards of care in various ...

When a robust long-time patient with chronic pain and heavy alcohol use declines precipitously after a complicated hospital course, her medical team must find a way to revive her old self.

Whether screening with blood-based early-detection tests for multiple cancers in addition to usual care has clinical utility is unknown. In a randomized, controlled trial conducted in England, we ...

Settlements of lawsuits against opioid manufacturers and other entities will shape drug policy for decades. Attention is needed to ensure that the funds will be dedicated to evidence-based activities.

To address drivers of poor child health, society should move from responding to health crises one symptom and one child at a time to reimagining ecosystems in ways that facilitate healthy development.

Gene therapy can spare people with sickle cell disease from recurrent vaso-occlusive crises, but for the populations that bear most of the global burden of disease, there’s no pathway to such treat ...

Acute metabolic acidosis is common in hospitalized patients owing to conditions such as shock, kidney disease, and diabetic ketoacidosis. While the underlying cause is being treated, it may be ...

Colonoscopy surveillance after the removal of adenomas is a cornerstone of the prevention of colorectal cancer. Guidelines in the United States and Europe have endorsed surveillance beginning at 3 ...

More evidence is needed to inform recommendations for intervals of colonoscopy surveillance after polyp removal. In this ongoing noninferiority trial conducted in eight European countries, we ...

Tambotatug pelitecan (known as Tam-Peli, a new antibody–drug conjugate that targets the immune-checkpoint molecule B7-H3) showed promising clinical efficacy in patients with relapsed extensive-stage ...

A 3-year-old boy with chemotherapy-resistant metastatic hepatoblastoma had complete regression lasting at least 12 months after two infusions of glypican-3–specific CAR T cells coexpressing ...