A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Inclusion Criteria

• •Written informed consent must be obtained from the parent or guardian prior to performing any study related procedures;
• •Patient must have a clinical diagnosis of GSD-II confirmed by endogenous GAA activity below normal in at least one tissue;
• •Patient must have a sibling with a clinical diagnosis of GSD-II confirmed by an endogenous GAA activity below normal in at least one tissue, who is eligible for participation in this study;
• •Patient must have a sibling with identical GAA mutations who is eligible for participation in this study;
• •Patient must have a sibling with evidence of different progression of GSD-II who is eligible for participation in this study;
• •The patient or his/her guardian(s) must have the ability to comply with the clinical protocol.