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

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 ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in ...

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 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.

A man from rural India presented with redness and blurry vision in one eye. Examination was notable for panuveitis and a live worm in the posterior segment; the worm was removed in a pars plana vit ...

The former members of the Advisory Committee on Immunization Practices consider paths forward for U.S. vaccine policy in the wake of their dismissal by the Secretary of Health and Human Services in ...

To prevent the spread of inaccurate information, academic and health care institutions will need to equip scientists and clinicians to engage effectively on nontraditional media platforms.

Understanding today’s corporatization of U.S. health care requires seeing it from a historical perspective, as a process that began with a change in the business model of care delivery in the 1920s.

A 27-year-old man with e-cigarette use presented with a 5-day history of fever, vomiting, diarrhea, dyspnea, and hemoptysis. A radiograph and CT scan of the chest showed patchy consolidations in bo ...

The extreme reductions that the Trump administration is making at the vital, mission-driven Centers for Disease Control and Prevention amount to unilateral disarmament against health hazards.

The rising tide of chronic kidney disease among patients with type 2 diabetes continues to challenge clinicians and health systems worldwide.1 Advances have been made in the management of chronic k ...