Genzyme's Drug for Rare Enzyme Deficiency Is Approved - New York Times: "Genzyme won federal approval yesterday to sell the first drug for Pompe disease, a rare inherited enzyme deficiency that destroys muscles and can kill infants by their first birthday.

The drug, Myozyme, is the fourth developed by Genzyme to treat a rare enzyme deficiency under the incentives provided by the federal government to develop medicines for so-called orphan diseases. "