The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent.
The patient has a confirmed GAA enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
Infant and toddler Pompe disease patients can be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) will not exceed 3 percent of the total blood volume during a period of 4 weeks and will not exceed 1 percent at any single time.
The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.
For patients previously treated with alglucosidase alfa the patient has received alglucosidase alfa for at least 6 months.

NCT01410890_inc_T2
Condition
GAA enzyme deficiency
NCT01410890_inc_T3
Qualifier
skin
NCT01410890_inc_T4
Qualifier
blood
NCT01410890_inc_T5
Qualifier
muscle tissue
NCT01410890_inc_T6
Multiplier
2
NCT01410890_inc_T7
Observation
GAA gene mutations
NCT01410890_inc_T8
Scope
skin, blood, or muscle tissue
NCT01410890_inc_T9
Condition
Pompe disease
NCT01410890_inc_T10
Person
Infant
NCT01410890_inc_T11
Person
toddler
NCT01410890_inc_T15
Drug
alglucosidase alfa
NCT01410890_inc_T16
Temporal
for at least 6 months