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

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

A 28-year-old woman was admitted to the hospital because of severe hypoxemia and right heart failure that had been caused by rapidly progressive pulmonary hypertension. A diagnosis was made.

Audio Interview from the New England Journal of Medicine — Interview with Tom Frieden on the role of the CDC and current threats to the U.S. public health infrastructure.