Genzyme Corp. (NASDAQ: GENZ) today announced that the results from its Late-Onset Treatment Study (LOTS) have been published in today's New England Journal of Medicine. The study was undertaken to evaluate the safety and efficacy of Myozyme (alglucosidase alfa) in older children and adults with Pompe disease.
“The study is an important contribution to the field of Pompe disease.”
The LOTS study was a randomized, double-blind, multi-national, placebo-controlled clinical trial that enrolled 90 older children and adults with Pompe disease. The study met its co-primary endpoints, demonstrating a beneficial effect of alglucosidase alfa on functional endurance and musculoskeletal strength as measured by the six-minute walk test, and on pulmonary function as measured by percent predicted forced vital capacity. Secondary endpoints of the study were supportive of these positive results.
"The data from LOTS demonstrated a positive effect of enzyme replacement therapy in older children and adults with Pompe disease," said Ans van der Ploeg, M.D., Ph.D., of Erasmus Medical Center in Rotterdam and the study's lead author. "The study is an important contribution to the field of Pompe disease."