FDAnews
www.fdanews.com/articles/80969-genzyme-begins-trial-of-myozyme-for-pompe-s-disease

GENZYME BEGINS TRIAL OF MYOZYME FOR POMPE'S DISEASE

September 16, 2005

Genzyme has initiated a clinical trial evaluating the safety and efficacy of Myozyme (alglucosidase alfa) in patients with late-onset Pompe's disease. The trial is designed to provide additional support for Myozyme's use.

Patients with Pompe's disease display a range of symptoms that correspond with the age of disease onset and the rate of disease progression. Progressive weakness in the muscles used for mobility, breathing and digestion are the most common symptoms. In infants, heart muscle is typically severely affected.

The late-onset trial is a randomized, double-blind, placebo-controlled study that will enroll at least 72 patients. It is being conducted at five sites in the U.S. and two in Europe. Participants will be assigned to receive intravenous infusions of either Myozyme at a dose of 20 mg/kg or a placebo every other week for 52 weeks.