T.O.4 Safety and efficacy results from a randomized, double-blind, placebo-controlled study of alglucosidase alfa for the treatment of Pompe disease in juveniles and adults

1 Institut de Myologie, Groupe Hospitalier Pitie-Salpetriere, Paris, France

2 University of Pittsburgh, Department of Neurology, Pittsburgh, United States

3 Genzyme Corporation, Cambridge, United States

4 Children’s National Medical Center, Washington, DC, United States

5 Washington University, School of Medicine, St. Louis, United States

6 Erasmus Medical Center, Rotterdam, The Netherlands

7 Washington University, School of Medicine, St. Louis, United States

8 Tower Hematology Oncology, Beverly Hills, United States

9 Mount Sinai School of Medicine, New York, United States