The medicine increases the activity of a deficient enzyme in certain patients with the condition.

If the UK leaves the EU in March without a deal, transport of insulin and other essential drugs could be delayed.

Compared to other drugs, therapeutics given breakthrough status receive FDA approval on the basis of weaker trial evidence, a study finds.

Frances Oldham Kelsey, a physician who halted use of a drug that caused birth defects in babies, has passed away at age 101.

A new estimate finds the pricetag for taking a drug through the approval process has steadily risen over the past few decades.

Researchers have developed a technique for determining the species of snake responsible for a bite by sequencing genetic material from the fang marks.

Tigers Forever, High Moon Over the Amazon, Earth from Space, and Medicine's Michelangelo

Researchers who studied stargazing dung beetles, opera-loving mice are among recipients of this year’s Ig Nobel Prizes.

The researcher who falsely claimed to treat human patients with their own stem cells is dismissed, but insists that he did perform the procedure on one patient.