BEIJING, April 29 (Xinhuanet) -- The U.S. Food and Drug Administration (FDA) has approved Myozyme, made by Genzyme Corp., as the first treatment for Pompe disease Friday.
Pompe disease, a genetic disorder, affects between 5,000 and 10,000 people worldwide, and is frequently fatal. Most babies born with the condition die from respiratory or heart complications before 18 months of age.
An inherited deficiency in the enzyme alpha-glucosidase causes the neuromuscular disease. The enzyme is responsible for breaking down glycogen, a form of sugar stored in the muscles. As a result, Pompe patients suffer a buildup of glycogen in the muscles, eventually weakening them.
Myozyme will bear the most serious warning label, cautioning patients of the risk of life-threatening allergic reactions.
It was approved in Europe earlier this year. Enditem
(Agencies)
