Families of infants with a rare and devastating metabolic disease could soon have their first chance at treatment.
That’s because the Food and Drug Administration has granted orphan drug status for a new medication that treats what’s known as galactosemia. The disease affects how the body metabolizes galactose, a simple sugar found in dairy products but also naturally produced by the human body. Infants with galactosemia have too much galactose in their blood, which can lead to serious complications.