There's a group of rare diseases in which babies are born missing one of 11 different enzymes our bodies use to break down fatty sugars in our blood.
Absent these enzymes, fat molecules accumulate in critical organs like the heart and liver, often claiming the lives of those afflicted while they are still children.
When the first drug to target one of these diseases came along in 1991 a medicine called Ceredase, which was used to treat Gaucher's disease regulators approved the new enzyme replacement on the basis of a small and short trial.
Read Full Article »