The FDA has begun soliciting feedback to inform the next version of the Prescription Drug User Fee Act. User fees remain ...

MASLD has become the most common chronic liver disease worldwide. The authors review the features of the disease as well as pharmacotherapies targeting the associated liver and ...

Malignant T-cell transformation after chimeric antigen receptor (CAR) T-cell therapy has been described, but the contribution of CAR integration to oncogenesis is not clear. Here we report a case o ...

A rare T-cell lymphoma after chimeric antigen receptor (CAR) T-cell therapy for myeloma was linked to CCR4 overexpression and vector integration, which highlights the need for monitoring after CAR ...

Dry eye is a common, painful ocular disturbance that can result from systemic inflammatory diseases, localized eye problems, or commonly used medications. Current treatments address symptoms, but a ...

A small-molecule drug proves its mettle in the treatment of spinal muscular atrophy, a disease amenable to intervention at the pre-mRNA level.

The benefits of women’s health research haven’t been limited to women. But actions by the Trump administration threaten to dismantle the hard-won progress in this area.

In 1979, China implemented the one-child family policy, which restricts many couples to a single offspring. The authors describe the policy's success in slowing population growth as well as its adv ...

Respiratory distress developed in a neonate after delivery at 32 weeks of gestation. Endotracheal intubation was unsuccessful, and he died 4 hours after delivery. A postmortem esophagram obtained a ...

To the Editor: In their article on the PROPEL2 trial, Savarirayan and colleagues (Feb. 27 issue)1 describe the results of a phase 2 dose-finding trial of infigratinib in children with achondroplasi ...

To the Editor: Cederholm and Bosaeus (July 11 issue)1 propose three causal subtypes of malnutrition: disease-related with and without underlying inflammation and “starvation due to inadequate ...

The author describes the scientific foundations of a clinical trial of a first-in-class small-molecule estrogen receptor degrader to treat patients with metastatic breast cancer.